◎ Sugi Atlas

Atlas / Gene / SCN4A

SCN4A

gene
On this page

Also known as Nav1.4HYPPSkM1

Summary

SCN4A (sodium voltage-gated channel alpha subunit 4, HGNC:10591) is a protein-coding gene on chromosome 17q23.3, encoding Sodium channel protein type 4 subunit alpha (P35499). Pore-forming subunit of Nav1.4, a voltage-gated sodium (Nav) channel that directly mediates the depolarizing phase of action potentials in excitable membranes.

Voltage-gated sodium channels are transmembrane glycoprotein complexes composed of a large alpha subunit with 24 transmembrane domains and one or more regulatory beta subunits. They are responsible for the generation and propagation of action potentials in neurons and muscle. This gene encodes one member of the sodium channel alpha subunit gene family. It is expressed in skeletal muscle, and mutations in this gene have been linked to several myotonia and periodic paralysis disorders.

Source: NCBI Gene 6329 — RefSeq curated summary.

At a glance

  • Gene–disease (curated): SCN4A-related myopathy, autosomal recessive (Definitive, ClinGen) — +12 more curated relationships
  • GWAS associations: 7
  • Clinical variants (ClinVar): 1,107 total — 40 pathogenic, 29 likely-pathogenic
  • Phenotypes (HPO): 213
  • Druggable target: yes — 24 molecules with ChEMBL bioactivity
  • MANE Select transcript: NM_000334

Identifiers

Gene identifiers

FieldValue
HGNC IDHGNC:10591
Approved symbolSCN4A
Namesodium voltage-gated channel alpha subunit 4
Location17q23.3
Locus typegene with protein product
StatusApproved
AliasesNav1.4, HYPP, SkM1
Ensembl geneENSG00000007314
Ensembl biotypeprotein_coding
OMIM603967
Entrez6329

Gene structure

Transcript identifiers

Ensembl transcripts: 3 — 2 retained_intron, 1 protein_coding

ENST00000435607, ENST00000581514, ENST00000584310

RefSeq mRNA: 1 — MANE Select: NM_000334 NM_000334

CCDS: CCDS45761

Canonical transcript exons

ENST00000435607 — 24 exons

ExonStartEnd
ENSE000003965296394536063945638
ENSE000007423206394282663943096
ENSE000007423426394467363944810
ENSE000007423486394704563947167
ENSE000007423556394939363949528
ENSE000007423576395142463951900
ENSE000007423616395926563959438
ENSE000007423646396119363961431
ENSE000007423696396446863964677
ENSE000007423736396648163966544
ENSE000007423756396802363968355
ENSE000013127626393855463941993
ENSE000013128066396367263963825
ENSE000013219806397256963972918
ENSE000016464546394500763945060
ENSE000016667386396610263966243
ENSE000017144086397235263972470
ENSE000017454636397213663972225
ENSE000017963806394374663943850
ENSE000024304846395716263957518
ENSE000024648026397172263971850
ENSE000024719416397116263971253
ENSE000025108266394789063948063
ENSE000035884466394861163948765

Expression profiles

Bgee: expression breadth ubiquitous, 153 present calls, max score 96.32.

FANTOM5 (CAGE): breadth tissue_specific, TPM avg 0.1171 / max 20.4757, expressed in 54 samples.

FANTOM5 promoters (1 alternative TSS)

Promoter IDTPM avgSamples expressed
1675360.117154

Top tissues by expression

270 total, by Bgee expression score (0-100, higher = more expressed):

TissueAnatomy IDExpression scoreQuality
hindlimb stylopod muscleUBERON:000425296.32gold quality
gastrocnemiusUBERON:000138895.59gold quality
skeletal muscle tissue of rectus abdominisUBERON:000451195.00gold quality
vastus lateralisUBERON:000137994.26gold quality
muscle of legUBERON:000138393.96gold quality
muscle organUBERON:000163093.78gold quality
quadriceps femorisUBERON:000137793.68gold quality
gluteal muscleUBERON:000200093.65gold quality
diaphragmUBERON:000110393.51gold quality
triceps brachiiUBERON:000150992.99gold quality
skeletal muscle tissueUBERON:000113492.78gold quality
skeletal muscle tissue of biceps brachiiUBERON:000450292.40gold quality
biceps brachiiUBERON:000150792.11gold quality
muscle tissueUBERON:000238586.64gold quality
deltoidUBERON:000147685.86gold quality
omental fat padUBERON:001041485.10gold quality
peritoneumUBERON:000235885.03gold quality
adipose tissue of abdominal regionUBERON:000780884.73gold quality
tibialis anteriorUBERON:000138581.60silver quality
adipose tissueUBERON:000101380.40gold quality
subcutaneous adipose tissueUBERON:000219080.16gold quality
connective tissueUBERON:000238479.19gold quality
body of tongueUBERON:001187674.15gold quality
right lobe of thyroid glandUBERON:000111973.38gold quality
endometrium epitheliumUBERON:000481172.82gold quality
left lobe of thyroid glandUBERON:000112072.06gold quality
cerebellar vermisUBERON:000472071.98gold quality
thyroid glandUBERON:000204671.45gold quality
tongueUBERON:000172371.12silver quality
tendon of biceps brachiiUBERON:000818869.99gold quality

Single-cell (SCXA)

Detected in 1 experiment(s), a significant marker in 0.

ExperimentMarker?Max mean expression
E-ANND-3no3.67

Regulation

Is transcription factor: no

Upstream regulators (CollecTRI, top): IL10, TNF

miRNA regulators (miRDB)

92 targeting SCN4A, top 30 by miRDB confidence (max_score; target_count = how many genes the miRNA targets in total — lower means more specific):

miRNAMax scoreAvg scoremiRNA target_count
HSA-MIR-8485100.0077.574731
HSA-MIR-4455100.0065.481587
HSA-MIR-5193100.0067.261744
HSA-MIR-4692100.0067.322066
HSA-MIR-4533100.0069.482758
HSA-MIR-607799.9968.042299
HSA-MIR-451499.9967.101870
HSA-MIR-4789-3P99.9970.752484
HSA-MIR-3667-3P99.9967.171636
HSA-MIR-499A-5P99.9870.791323
HSA-MIR-4650-5P99.9864.69999
HSA-MIR-4789-5P99.9870.762721
HSA-MIR-6825-5P99.9669.813431
HSA-MIR-185-3P99.9567.011743
HSA-MIR-329-3P99.9166.561234
HSA-MIR-362-3P99.9166.381267
HSA-MIR-4731-5P99.8967.232537
HSA-MIR-477999.8666.501583
HSA-MIR-5003-3P99.8569.292517
HSA-MIR-76599.8468.242442
HSA-MIR-205299.7969.372031
HSA-MIR-3150A-3P99.7664.441640
HSA-MIR-6763-5P99.7664.681767
HSA-MIR-431999.7669.832586
HSA-MIR-3934-3P99.7665.511351
HSA-MIR-6764-5P99.7567.892304
HSA-MIR-3617-5P99.7569.411968
HSA-MIR-64199.7569.351975
HSA-MIR-11181-3P99.7566.382205
HSA-MIR-120099.7170.421838

Literature-anchored findings (GeneRIF, showing 40)

  • identified the Met1592Val mutation of SCN4A in an affected descendent of the original normokalemic periodic paralysis (normoKPP) family (PMID:12210802)
  • V1442E mutation in SCN4A defines a novel disease mechanism and a novel phenotype with myasthenic features. (PMID:12766226)
  • charged residues in the DIII-DIV linker may interact with structures that control slow inactivation in the voltage-gated sodium channel (hNa(V)1.4) (nav1.4) (PMID:14527681)
  • Cold exacerbation in two paramyotonia congenita mutant NaV1.4 channels R1448C and T1313M arises from a combination of biophysical defects that increase hyperexcitability at low temperatures. (PMID:15318338)
  • fast inactivation of the Na(+) channel involves both S4-S5 loops in D3 and D4 in a cooperative manner and D3/S4-S5 also plays an important role in activation and deactivation (PMID:15459238)
  • A novel SCN4A Arg672Cys mutation and a known CACNL1A3 Arg528His mutation were identified in in Korean hypokalemic periodic paralysis patients (PMID:15482957)
  • 3 new mutations were found in codon 675: an AA substitution of a highly conserved Arg to either a Gly, a Gln or a Trp. Hypokalemic periodic paralysis is caused by mutations affecting nearby codons as well as the change of an Arg into another AA. (PMID:15596759)
  • Fast inactivation and deactivation gating were compared between wild-type human voltage-gated skeletal muscle sodium channel (hNaV1.4) and potassium-aggravated myotonia (PAM) mutations G1306A, G1306E, and G1306V. (PMID:16392038)
  • Method in which voltage dependence of voltage-gated ion channel blockers can be compared using a protocol in which voltage error is compensated for in real time. (PMID:16506889)
  • A missense mutation is responsible for the hypokalemic periodic paralysis in a Chinese family. (PMID:16870577)
  • We identified a novel hypokallemic periodic paralysis type 2 mutation that neutralizes an arginine residue in DIII-S4 (R1132Q), and studied its functional consequences in HEK cells transfected with the human SCN4A cDNA. (PMID:16890191)
  • Deactivation gating relies on charged ammino-acids in DIIIS4 domain. (PMID:17151947)
  • present study has revealed a heterozygous A1481D (c.4442 C>A) missense mutation in the SCN4A gene, which was identified in family members affected with cold-aggravated myotonia in a large German kindred (PMID:17212350)
  • Warm-up phenomenon in myotonia associated with the V445M sodium channel mutation. (PMID:17334961)
  • HyperPP was suspected by means of clinical features and provocative testing, it was sequential genetic analysis that corroborated the diagnosis and identified a de novo mutation in the SCN4A gene. (PMID:17804458)
  • These results indicate that, in these paramyotonia congenita patients, mutant and wild-type sodium channels respond equally to cold exposure. (PMID:17823953)
  • Consistent with other NaV1.4 mutations associated with a paralytic phenotype, the P1158S mutation disrupts slow inactivation. The unique temperature sensitivity of the channel defect may contribute to the unusual clinical phenotype. (PMID:17898326)
  • A large cohort of French-Canadians with a founder SCN4A mutation causing painful cold-induced myotonia underlines the phenotypic heterogeneity of SCN4A mutations with variants in other genes such as CLCN1 that are likely to modulate clinical expression. (PMID:17998485)
  • In addition to Val-781-Ile and Met1592Val, the mutation g2101a (Arg675Gln) may be the novel mutation of SCN4A genes in Chinese patients with normoKPP. (PMID:18046642)
  • State- and use-dependent block of muscle Nav1.4 voltage-gated Na+ channel isoforms by ranolazine is reported. (PMID:18079277)
  • This study identifies two new mutations(R1448L and L1436P) confirms SCN4A as a common cause of paramyotonia congenita in the UK. (PMID:18166706)
  • The mechanism of calmodium modulation of Nav1.4 expression an function were studied. (PMID:18270170)
  • HyperPP is caused by mutations in the SCN4A gene that encodes the subunit of the NavCh Nav1.4 in skeletal muscles. (PMID:18281721)
  • CLCN1 and SCN4A mutation occurrence is associated with non-dystrophic myotonia. (PMID:18337730)
  • study investigated effects of paramyotonia congenita mutations F1473S and F1705I on gating of skeletal muscle Na+ channels; results suggest that the DIV S4-S5 linker mutation F1473S promotes the hyperpolarized position of DIVS4 to accelerate recovery (PMID:18690054)
  • Results decribe the Thr704Met point mutation in the SCN4A gene in hyperkalemic periodic paralysis. (PMID:18726720)
  • A novel dominant mutation of the Nav1.4 alpha-subunit domain I leading to sodium channel myotonia. (PMID:19015483)
  • These observations of one family confirmed that tubular aggregates were associated with T704M mutations of SCN4A in paralysis periodica paramyotonica. (PMID:19077043)
  • All SCN4A mutations affected arginine residues, consistent with the gating pore cation leak hypothesis of hypokalemic periodic paralysis. Arginine mutations in S4 segments underlie 90% of hypokalemic periodic paralysis cases. (PMID:19118277)
  • The clinical guidelines proposed may help clinicians working in outpatient clinics to perform a focused genetic analysis of either CLCN1 or SCN4A. (PMID:19211598)
  • Myoblastic cells from patients with myopathy caused by mutation SCN4A Metl592Val had a weaker ability of developing into the myotubules. (PMID:19290024)
  • This study has identified a new mutation of PAM with a severe phenotype and emphasizes the importance of the C-terminus for fast inactivation of the sodium channel(nav1.4, Q1633E). (PMID:19347921)
  • confirmed missense mutation in SCN4A in myotonia congenita (PMID:19840739)
  • electrostatic network interactions between S2 and other transmembrane segments within Na(v)1.4D4 are similar to but not identical to those proposed for K+ channels (PMID:19881885)
  • This study describes the first cases of homozygosity for two missense mutations in the SCN4A gene which increases severity of muscle channelopathies. (PMID:19882638)
  • study showed that a paroxysmal extreme pain disorder mutation in the Nav1.7 channel, a paramyotonia congenita mutation in the Nav1.4 channel & a long-QT3/SIDS mutation in the NAV1.5 channel all increased the amplitude of resurgent sodium currents (PMID:20038812)
  • Severe neonatal episodic laryngospasm is a new phenotype caused by a sodium channelopathy, which can be alleviated by channel blockers. (PMID:20713951)
  • study detected the SCN4A R672H mutation in one hypokalemic periodic paralysis Turkish family (PMID:21043388)
  • Anthopleurin elicited opposing effects on the gating mode, kinetics and charge immobilized during open- versus closed-state fast inactivation of Nav1.4 channels. (PMID:21099342)
  • ranolazine interacts with the open state and stabilizes the inactivated state(s) of Na(v)1.4 channels, causes voltage- and use-dependent block of I(Na) and suppresses persistent I(Na) (PMID:21317558)

Cross-species orthologs

2 orthologs

OrganismSymbolGene ID
mus_musculusScn4aENSMUSG00000001027
rattus_norvegicusScn4aENSRNOG00000012134

Paralogs (26): CACNA1G (ENSG00000006283), CACNA1S (ENSG00000081248), CACNA1I (ENSG00000100346), CACNA1F (ENSG00000102001), NALCN (ENSG00000102452), SCN2A (ENSG00000136531), SCN7A (ENSG00000136546), CACNA1A (ENSG00000141837), SCN1A (ENSG00000144285), CACNA1B (ENSG00000148408), CACNA1C (ENSG00000151067), CATSPER3 (ENSG00000152705), SCN3A (ENSG00000153253), CACNA1D (ENSG00000157388), TPCN2 (ENSG00000162341), CATSPER2 (ENSG00000166762), SCN11A (ENSG00000168356), SCN9A (ENSG00000169432), CATSPER1 (ENSG00000175294), SCN5A (ENSG00000183873), SCN10A (ENSG00000185313), TPCN1 (ENSG00000186815), CATSPER4 (ENSG00000188782), CACNA1H (ENSG00000196557), SCN8A (ENSG00000196876), CACNA1E (ENSG00000198216)

Protein

Protein identifiers

Sodium channel protein type 4 subunit alpha — P35499 (reviewed: P35499)

Alternative names: SkM1, Sodium channel protein skeletal muscle subunit alpha, Sodium channel protein type IV subunit alpha, Voltage-gated sodium channel subunit alpha Nav1.4

All UniProt accessions (1): P35499

UniProt curated annotations — full annotation on UniProt →

Function. Pore-forming subunit of Nav1.4, a voltage-gated sodium (Nav) channel that directly mediates the depolarizing phase of action potentials in excitable membranes. Navs, also called VGSCs (voltage-gated sodium channels) or VDSCs (voltage-dependent sodium channels), operate by switching between closed and open conformations depending on the voltage difference across the membrane. In the open conformation they allow Na(+) ions to selectively pass through the pore, along their electrochemical gradient. The influx of Na+ ions provokes membrane depolarization, initiating the propagation of electrical signals throughout cells and tissues. Highly expressed in skeletal muscles, Nav1.4 generates the action potential crucial for muscle contraction.

Subunit / interactions. The Nav1.4 voltage-gated sodium channel consists of an ion-conducting alpha subunit SCN4A which is functional on its own and a regulatory beta subunit SCN1B. SCN1B strongly enhances the presence of SCN4A at the cell surface. SCN1B is also required for rapid channel inactivation and recovery after inactivation. It prevents the decrease of channel activity in response to repetitive, high-frequency depolarizations. Interacts with the syntrophins SNTA1, SNTB1 and SNTB2 (via PDZ domain); probably links SCN4A to the actin cytoskeleton and the extracellular matrix via the dystrophin-associated protein complex and regulates its localization in muscle cells. Interacts with TMEM233; probable regulator of the channel.

Subcellular location. Cell membrane.

Disease relevance. Paramyotonia congenita (PMC) [MIM:168300] An autosomal dominant channelopathy characterized by myotonia, increased by exposure to cold, intermittent flaccid paresis, not necessarily dependent on cold or myotonia, lability of serum potassium, non-progressive nature and lack of atrophy or hypertrophy of muscles. In some patients, myotonia is not increased by cold exposure (paramyotonia without cold paralysis). Patients may have a combination phenotype of PMC and HYPP. The disease is caused by variants affecting the gene represented in this entry. Periodic paralysis hypokalemic 2 (HOKPP2) [MIM:613345] An autosomal dominant disorder manifested by episodic flaccid generalized muscle weakness associated with falls of serum potassium levels. The disease is caused by variants affecting the gene represented in this entry. Periodic paralysis hyperkalemic (HYPP) [MIM:170500] An autosomal dominant channelopathy characterized by episodic flaccid generalized muscle weakness associated with high levels of serum potassium. Concurrence of myotonia is found in HYPP patients. The disease is caused by variants affecting the gene represented in this entry. Periodic paralysis normokalemic (NKPP) [MIM:170500] A disorder closely related to hyperkalemic periodic paralysis, but marked by a lack of alterations in potassium levels during attacks of muscle weakness. The disease is caused by variants affecting the gene represented in this entry. Myotonia SCN4A-related (MYOSCN4A) [MIM:608390] A phenotypically highly variable myotonia aggravated by potassium loading, and sometimes by cold. Myotonia is characterized by sustained muscle tensing that prevents muscles from relaxing normally. It causes muscle stiffness that can interfere with movement. In some people the stiffness is very mild, while in other cases it may be severe enough to interfere with walking, running, and other activities of daily life. Myotonia SCN4A-related includes myotonia permanens and myotonia fluctuans. In myotonia permanens, the myotonia is generalized and there is a hypertrophy of the muscle, particularly in the neck and the shoulder. Attacks of severe muscle stiffness of the thoracic muscles may be life threatening due to impaired ventilation. In myotonia fluctuans, the muscle stiffness may fluctuate from day to day, provoked by exercise. The disease is caused by variants affecting the gene represented in this entry. Myasthenic syndrome, congenital, 16 (CMS16) [MIM:614198] A form of congenital myasthenic syndrome, a group of disorders characterized by failure of neuromuscular transmission, including pre-synaptic, synaptic, and post-synaptic disorders that are not of autoimmune origin. Clinical features are easy fatigability and muscle weakness. CMS16 is characterized by fatigable generalized weakness and recurrent attacks of respiratory and bulbar paralysis since birth. The fatigable weakness involves lid-elevator, external ocular, facial, limb and truncal muscles and an decremental response of the compound muscle action potential on repetitive stimulation. The disease is caused by variants affecting the gene represented in this entry. Congenital myopathy 22A, classic (CMYO22A) [MIM:620351] A form of congenital myopathy, a clinically and genetically heterogeneous group of muscle disorders characterized by hypotonia and muscle weakness apparent at birth, and specific pathological features on muscle biopsy. CMYO22A is an autosomal recessive form characterized by fetal hypokinesia, polyhydramnios, and severe neonatal hypotonia associated with respiratory insufficiency. Affected individuals who survive the neonatal period have delayed motor development, difficulty walking, proximal muscle weakness of the upper and lower limbs, facial and neck muscle weakness, easy fatigability, and mild limb contractures or foot deformities. The disease is caused by variants affecting the gene represented in this entry. Congenital myopathy 22B, severe fetal (CMYO22B) [MIM:620369] A severe congenital myopathy, a clinically and genetically heterogeneous group of muscle disorders characterized by hypotonia and muscle weakness apparent at birth, and specific pathological features on muscle biopsy. CMYO22B is an autosomal recessive form characterized by onset in utero. Affected individuals show fetal akinesia, and develop fetal hydrops with pulmonary hypoplasia, severe joint contractures, and generalized muscle hypoplasia. Death occurs in utero or soon after birth. The disease is caused by variants affecting the gene represented in this entry.

Activity regulation. The channel is inhibited by tetrodotoxin and saxitoxin. Inhibited by the conotoxin GVIIJ.

Domain organisation. The sequence contains 4 internal repeats, each with 5 hydrophobic segments (S1, S2, S3, S5, S6) and one positively charged segment (S4). Segments S4 are probably the voltage-sensors and are characterized by a series of positively charged amino acids at every third position.

Similarity. Belongs to the sodium channel (TC 1.A.1.10) family. Nav1.4/SCN4A subfamily.

RefSeq proteins (1): NP_000325* (*=MANE)

Domains & families (InterPro)

IDNameType
IPR001696Na_channel_asuFamily
IPR005821Ion_trans_domDomain
IPR008052Na_channel_a4su_mammalFamily
IPR010526Na_trans_assoc_domDomain
IPR027359Volt_channel_dom_sfHomologous_superfamily
IPR043203VGCC_Ca_NaFamily
IPR044564Na_chnl_inactivation_gateDomain
IPR058542IQ_SCN5A_CDomain

Pfam: PF00520, PF06512, PF24609

Catalyzed reactions (Rhea), 1 shown:

  • Na(+)(in) = Na(+)(out) (RHEA:34963)

UniProt features (267 total): sequence variant 70, helix 68, topological domain 29, transmembrane region 24, strand 21, turn 11, glycosylation site 11, region of interest 6, disulfide bond 6, sequence conflict 5, compositionally biased region 5, intramembrane region 4, repeat 4, chain 1, domain 1, site 1

Structure

Experimental structures (PDB)

3 structures.

PDBMethodResolution (Å)
6MBAX-RAY DIFFRACTION1.8
6AGFELECTRON MICROSCOPY3.2
6MC9X-RAY DIFFRACTION3.3

Predicted structure (AlphaFold)

ModelpLDDTFraction very-high
AF-P35499-F173.090.20

Functional residue map

Curated UniProt residues grouped by drug-discovery relevance — catalytic, ligand-binding, modification, and mutation-validated positions. Source: UniProtKB sequence features.

Catalytic / active sites (1): 407 (important for inhibition by tetrodotoxin)

Disulfide bonds (6): 280–360, 369–375, 731–737, 769–778, 1189–1209, 1553–1568

Glycosylation sites (11): 214, 288, 291, 297, 303, 315, 321, 333, 362, 1191, 1205

Function

Pathways and Gene Ontology

Reactome pathways

8 pathways

IDPathway
R-HSA-445095Interaction between L1 and Ankyrins
R-HSA-5576892Phase 0 - rapid depolarisation
R-HSA-1266738Developmental Biology
R-HSA-373760L1CAM interactions
R-HSA-397014Muscle contraction
R-HSA-422475Axon guidance
R-HSA-5576891Cardiac conduction
R-HSA-9675108Nervous system development

MSigDB gene sets: 478 (showing top): GOBP_MEMBRANE_DEPOLARIZATION, YAGI_AML_WITH_INV_16_TRANSLOCATION, GOBP_CIRCULATORY_SYSTEM_PROCESS, GAANYNYGACNY_UNKNOWN, GOBP_SODIUM_ION_TRANSMEMBRANE_TRANSPORT, GOBP_MEMBRANE_DEPOLARIZATION_DURING_ACTION_POTENTIAL, GOBP_MULTICELLULAR_ORGANISMAL_MOVEMENT, MODULE_16, GOBP_SKELETAL_MUSCLE_CONTRACTION, CAGCTG_AP4_Q5, GOBP_MONOATOMIC_CATION_TRANSPORT, GOBP_REGULATION_OF_STRIATED_MUSCLE_CONTRACTION, GOBP_REGULATION_OF_MUSCLE_CONTRACTION, GOBP_MUSCLE_CONTRACTION, GOBP_CARDIAC_MUSCLE_CELL_CONTRACTION

GO Biological Process (9): sodium ion transport (GO:0006814), muscle contraction (GO:0006936), sodium ion transmembrane transport (GO:0035725), cardiac muscle cell action potential involved in contraction (GO:0086002), regulation of skeletal muscle contraction by action potential (GO:0100001), action potential (GO:0001508), monoatomic ion transport (GO:0006811), monoatomic ion transmembrane transport (GO:0034220), transmembrane transport (GO:0055085)

GO Molecular Function (5): voltage-gated sodium channel activity (GO:0005248), monoatomic ion channel activity (GO:0005216), monoatomic cation channel activity (GO:0005261), sodium channel activity (GO:0005272), protein binding (GO:0005515)

GO Cellular Component (4): voltage-gated sodium channel complex (GO:0001518), plasma membrane (GO:0005886), membrane (GO:0016020), monoatomic ion channel complex (GO:0034702)

Reactome top-level categories

Rollup of top-6 pathways:

CategoryPathways
L1CAM interactions1
Cardiac conduction1
Axon guidance1
Nervous system development1
Muscle contraction1
Developmental Biology1

GO top-level categories

Rollup of top GO terms by namespace:

CategoryTerms
transport2
metal ion transport1
muscle system process1
sodium ion transport1
monoatomic cation transmembrane transport1
cardiac muscle cell action potential1
cardiac muscle cell contraction1
action potential1
skeletal muscle contraction1
regulation of skeletal muscle contraction1
regulation of membrane potential1
monoatomic ion transport1
transmembrane transport1
cellular process1
sodium channel activity1
monoatomic ion transmembrane transporter activity1
channel activity1
monoatomic ion channel activity1
monoatomic cation transmembrane transporter activity1
monoatomic cation channel activity1
sodium ion transmembrane transporter activity1
binding1
sodium channel complex1
plasma membrane protein complex1
membrane1
cell periphery1
cellular anatomical structure1
transmembrane transporter complex1

Protein interactions and networks

STRING

1478 interactions, top by confidence (×1000):

Protein AProtein BPartner UniProtScore
SCN4ACLCN1P35523946
SCN4ANAV1Q8NEY1899
SCN4ACALM1P02593864
SCN4ASCN3BQ9NY72823
SCN4AKCNJ2P48049809
SCN4ACALML3P27482809
SCN4ACALML5Q9NZT1809
SCN4ACALML6Q8TD86803
SCN4ACALML4Q96GE6803
SCN4AKCNE3Q9Y6H6799
SCN4ASCN1BQ07699795
SCN4ASCN4BQ8IWT1791
SCN4ADOK7Q18PE1782
SCN4ACOLQQ9Y215781
SCN4AKCNH2Q12809741

IntAct

135 interactions, top by confidence:

ABTypeScore
NEURL4APBB1psi-mi:“MI:0914”(association)0.530
SCN4ASCN1Bpsi-mi:“MI:0407”(direct interaction)0.520
SCN4ADLG4psi-mi:“MI:0407”(direct interaction)0.440
SCN4ADLG1psi-mi:“MI:0407”(direct interaction)0.440
SCN4AMAGI3psi-mi:“MI:0407”(direct interaction)0.440
SCN4ASNX27psi-mi:“MI:0407”(direct interaction)0.440
SCN4ADLG2psi-mi:“MI:0407”(direct interaction)0.440
SCN4AMAGI2psi-mi:“MI:0407”(direct interaction)0.440
SCN4APDZD2psi-mi:“MI:0407”(direct interaction)0.440
SCN4ASYNJ2BPpsi-mi:“MI:0407”(direct interaction)0.440
SCN4ASNTB1psi-mi:“MI:0407”(direct interaction)0.440
PDZD7SCN4Apsi-mi:“MI:0407”(direct interaction)0.440
DLG3SCN4Apsi-mi:“MI:0407”(direct interaction)0.440
MAST2SCN4Apsi-mi:“MI:0407”(direct interaction)0.440
SCN4ASNTG1psi-mi:“MI:0407”(direct interaction)0.440
PTPN3SCN4Apsi-mi:“MI:0407”(direct interaction)0.440
IL16SCN4Apsi-mi:“MI:0407”(direct interaction)0.440
SCN4ASNTG2psi-mi:“MI:0407”(direct interaction)0.440
SCN4ASNTA1psi-mi:“MI:0407”(direct interaction)0.440
SCN4ATAX1BP3psi-mi:“MI:0407”(direct interaction)0.440
SCN4APDZK1psi-mi:“MI:0407”(direct interaction)0.440
MAGI1SCN4Apsi-mi:“MI:0407”(direct interaction)0.440
SCN4ADLG3psi-mi:“MI:0407”(direct interaction)0.440
SCN4ASCRIBpsi-mi:“MI:0407”(direct interaction)0.440

BioGRID (91): SCN4A (Proximity Label-MS), RPL10A (Proximity Label-MS), DNAJC3 (Proximity Label-MS), SCN4A (Proximity Label-MS), HIST1H1A (Proximity Label-MS), XIRP1 (Affinity Capture-MS), FZD2 (Affinity Capture-MS), GLRB (Affinity Capture-MS), SLC15A4 (Affinity Capture-MS), GPR126 (Affinity Capture-MS), PIGO (Affinity Capture-MS), GPAA1 (Affinity Capture-MS), TMPPE (Affinity Capture-MS), CYB5R1 (Affinity Capture-MS), FAM69A (Affinity Capture-MS)

ESM2 similar proteins: A2APX8, A2ASI5, B1AWN6, C9D7C2, D0E0C2, O08562, O35505, O46669, O73700, O88420, O88457, P02719, P04774, P04775, P08104, P0DMA5, P15381, P15389, P15390, P22002, P27732, P35498, P35499, P35500, Q01815, Q07652, Q13936, Q14524, Q15858, Q20JQ7, Q28371, Q28644, Q2XVR3, Q2XVR4, Q2XVR5, Q2XVR6, Q2XVR7, Q62205, Q62968, Q6QIY3

Diamond homologs: A2APX8, A2ASI5, B1AWN6, B1AYL1, D0E0C2, F1LQQ7, O00555, O08562, O46669, O73705, O73706, O73707, O88420, O88457, P02719, P04774, P04775, P08104, P0DMA5, P15389, P15390, P27884, P35498, P35499, P35500, P54282, P56699, P97445, Q01118, Q02789, Q05973, Q14524, Q15858, Q15878, Q20JQ7, Q28371, Q28644, Q2XVR3, Q2XVR4, Q2XVR5

SIGNOR signaling

10 interactions.

AEffectBMechanism
SCN4A“up-regulates quantity”sodium(1+)relocalization
FGF13“down-regulates activity”SCN4Abinding
FGF12“down-regulates activity”SCN4Abinding
FGF14“down-regulates activity”SCN4Abinding
FGF11“down-regulates activity”SCN4Abinding
SCN4Aup-regulatesAction_potential
NEDD4L“down-regulates quantity by destabilization”SCN4Aubiquitination
TNF“up-regulates quantity by expression”SCN4A“transcriptional regulation”
TNF“up-regulates activity”SCN4A
IL10“down-regulates quantity by repression”SCN4A“transcriptional regulation”

Enriched among interaction partners

Reactome pathways and GO biological processes over-represented among this gene’s 91 IntAct physical interaction partners (hypergeometric vs the genome-wide background, BH-FDR, gene-set size 15–500, ranked by fold). A functional readout of the neighbourhood — distinct from this gene’s own memberships above, and biased toward well-studied / hub proteins, so read it as themes rather than proof.

Reactome pathways:

PathwayPartnersFoldFDR
Ras activation upon Ca2+ influx through NMDA receptor546.8×3e-06
Unblocking of NMDA receptors, glutamate binding and activation544.6×3e-06
Negative regulation of NMDA receptor-mediated neuronal transmission544.6×3e-06
Assembly and cell surface presentation of NMDA receptors1041.6×3e-12
Dopamine Neurotransmitter Release Cycle540.7×4e-06
Long-term potentiation539.0×5e-06
Neurexins and neuroligins1135.5×2e-12
Protein-protein interactions at synapses730.5×2e-07

GO biological processes:

GO termPartnersFoldFDR
establishment or maintenance of epithelial cell apical/basal polarity1172.6×7e-16
protein localization to synapse652.2×2e-07
receptor clustering749.6×2e-08
regulation of postsynaptic membrane neurotransmitter receptor levels739.4×7e-08
cell-cell adhesion1011.5×1e-06
protein-containing complex assembly810.3×6e-05
regulation of small GTPase mediated signal transduction58.2×7e-03
chemical synaptic transmission76.2×4e-03

Disease & clinical

Clinical variants and AI predictions

ClinVar

1107 variants total. Per-class counts are floors (≥ shown; pagination cap):

ClassificationCount (floor)
Pathogenic40
Likely pathogenic29
Uncertain significance556
Likely benign316
Benign49

Top pathogenic / likely-pathogenic (30)

Variant IDHGVSClassification
1032988NM_000334.4(SCN4A):c.685del (p.Thr229fs)Pathogenic
1074599NM_000334.4(SCN4A):c.608T>A (p.Met203Lys)Pathogenic
1352149NM_000334.4(SCN4A):c.918G>A (p.Trp306Ter)Pathogenic
1362468NM_000334.4(SCN4A):c.1775C>T (p.Thr592Ile)Pathogenic
1420693NM_000334.4(SCN4A):c.1800C>G (p.Tyr600Ter)Pathogenic
143201NM_000334.4(SCN4A):c.3404G>A (p.Arg1135His)Pathogenic
1453704NM_000334.4(SCN4A):c.584G>A (p.Trp195Ter)Pathogenic
1457445NC_000017.10:g.(?62024385)(62034898_?)delPathogenic
1458366NM_000334.4(SCN4A):c.1249C>T (p.Arg417Ter)Pathogenic
1980672NM_000334.4(SCN4A):c.1925G>A (p.Trp642Ter)Pathogenic
2001963NM_000334.4(SCN4A):c.1495del (p.Asp499fs)Pathogenic
2017990NM_000334.4(SCN4A):c.1905C>A (p.Tyr635Ter)Pathogenic
21151NM_000334.4(SCN4A):c.2014C>T (p.Arg672Cys)Pathogenic
2117553NM_000334.4(SCN4A):c.1049_1053del (p.Tyr349_Phe350insTer)Pathogenic
2133553NM_000334.4(SCN4A):c.3397del (p.Ala1133fs)Pathogenic
221261NM_000334.4(SCN4A):c.1320T>G (p.Asn440Lys)Pathogenic
221263NM_000334.4(SCN4A):c.4343G>C (p.Arg1448Pro)Pathogenic
2501680NM_000334.4(SCN4A):c.1144C>A (p.Pro382Thr)Pathogenic
2501683NM_000334.4(SCN4A):c.1123T>C (p.Cys375Arg)Pathogenic
2698109NM_000334.4(SCN4A):c.1641G>A (p.Trp547Ter)Pathogenic
2736637NM_000334.4(SCN4A):c.3937A>G (p.Thr1313Ala)Pathogenic
2789589NM_000334.4(SCN4A):c.1125C>A (p.Cys375Ter)Pathogenic
2862383NM_000334.4(SCN4A):c.4354_4369del (p.Leu1452fs)Pathogenic
2865882NM_000334.4(SCN4A):c.1331C>A (p.Ala444Asp)Pathogenic
3707358NM_000334.4(SCN4A):c.501del (p.Tyr168fs)Pathogenic
4725146NM_000334.4(SCN4A):c.1286del (p.Val429fs)Pathogenic
4727872NM_000334.4(SCN4A):c.971G>A (p.Trp324Ter)Pathogenic
4728986NM_000334.4(SCN4A):c.14_18del (p.Ser5fs)Pathogenic
5910NM_000334.4(SCN4A):c.1333G>A (p.Val445Met)Pathogenic
5911NM_000334.4(SCN4A):c.2006G>A (p.Arg669His)Pathogenic

SpliceAI

5238 predictions. Top by Δscore:

VariantEffectΔscore
17:63943740:CTGTA:Cdonor_loss1.0000
17:63943742:GTACC:Gdonor_loss1.0000
17:63943743:TACC:Tdonor_loss1.0000
17:63943744:A:Cdonor_loss1.0000
17:63943745:C:CAdonor_loss1.0000
17:63943842:CCCCC:Cacceptor_gain1.0000
17:63943843:CCCCC:Cacceptor_gain1.0000
17:63943844:CCC:Cacceptor_gain1.0000
17:63943845:CC:Cacceptor_gain1.0000
17:63943845:CCC:Cacceptor_gain1.0000
17:63943846:CC:Cacceptor_gain1.0000
17:63943846:CCTA:Cacceptor_loss1.0000
17:63943847:C:CCacceptor_gain1.0000
17:63943847:C:Tacceptor_gain1.0000
17:63943847:CT:Cacceptor_loss1.0000
17:63943851:G:GCacceptor_gain1.0000
17:63943853:A:Cacceptor_gain1.0000
17:63944667:A:ACdonor_gain1.0000
17:63944668:C:CCdonor_gain1.0000
17:63944671:A:ACdonor_gain1.0000
17:63944671:ATCTT:Adonor_gain1.0000
17:63944806:TCCTT:Tacceptor_gain1.0000
17:63944807:CCTTC:Cacceptor_gain1.0000
17:63944808:CTT:Cacceptor_gain1.0000
17:63944809:TT:Tacceptor_gain1.0000
17:63944809:TTC:Tacceptor_loss1.0000
17:63944810:TCTGT:Tacceptor_loss1.0000
17:63944811:C:CCacceptor_gain1.0000
17:63944811:C:CGacceptor_loss1.0000
17:63944812:T:Cacceptor_loss1.0000

AlphaMissense

12284 scored. Top likely-pathogenic:

VariantProtein changeam_pathogenicity
17:63941167:G:CF1705L1.000
17:63941167:G:TF1705L1.000
17:63941168:A:GF1705S1.000
17:63941169:A:GF1705L1.000
17:63941380:G:CF1634L1.000
17:63941380:G:TF1634L1.000
17:63941382:A:GF1634L1.000
17:63941412:A:GW1624R1.000
17:63941412:A:TW1624R1.000
17:63941489:A:GL1598P1.000
17:63941509:G:CN1591K1.000
17:63941509:G:TN1591K1.000
17:63941657:A:GL1542P1.000
17:63941668:C:AW1538C1.000
17:63941668:C:GW1538C1.000
17:63941670:A:GW1538R1.000
17:63941670:A:TW1538R1.000
17:63941810:A:GL1491P1.000
17:63941816:A:GL1489P1.000
17:63941830:G:CN1484K1.000
17:63941830:G:TN1484K1.000
17:63941837:A:GL1482P1.000
17:63941837:A:TL1482H1.000
17:63941843:G:CP1480R1.000
17:63941843:G:TP1480H1.000
17:63941858:A:GL1475P1.000
17:63941858:A:TL1475H1.000
17:63941863:G:CF1473L1.000
17:63941863:G:TF1473L1.000
17:63941865:A:GF1473L1.000

dbSNP variants (sampled 300 via entrez): RS1000005658 (17:63946605 G>A), RS1000325710 (17:63946346 A>G), RS1000390670 (17:63952164 G>T), RS1000492319 (17:63967218 T>C), RS1000526803 (17:63950482 T>A), RS1000581227 (17:63956099 C>A), RS1000613205 (17:63941280 T>A,C), RS1000630747 (17:63944938 G>A,C), RS1000784591 (17:63967361 C>A,G,T), RS1001000237 (17:63945502 G>A), RS1001030620 (17:63955789 T>G), RS1001051115 (17:63961396 A>G), RS1001100307 (17:63961935 C>T), RS1001101747 (17:63945860 A>C), RS1001218305 (17:63940390 A>T)

Disease associations

OMIM: gene MIM:603967 | disease phenotypes: MIM:170500, MIM:168300, MIM:613345, MIM:614198, MIM:608390, MIM:170400, MIM:620351, MIM:620369, MIM:601462, MIM:255300, MIM:617468, MIM:208150

GenCC curated gene-disease

DiseaseClassificationInheritance
SCN4A-related myopathy, autosomal recessiveDefinitiveAutosomal recessive
paramyotonia congenita of Von EulenburgDefinitiveAutosomal dominant
hyperkalemic periodic paralysisDefinitiveAutosomal dominant
congenital myasthenic syndrome 16StrongAutosomal recessive
congenital myopathyStrongAutosomal recessive
hypokalemic periodic paralysis, type 2StrongAutosomal dominant
potassium-aggravated myotoniaStrongAutosomal dominant
congenital myopathy 22A, classicStrongAutosomal recessive
hypokalemic periodic paralysisSupportiveAutosomal dominant
postsynaptic congenital myasthenic syndromeSupportiveAutosomal recessive
myotonia fluctuansSupportiveAutosomal dominant
myotonia permanensSupportiveAutosomal dominant
acetazolamide-responsive myotoniaSupportiveAutosomal dominant

ClinGen Gene-Disease Validity (1)

Expert-panel classifications — Definitive > Strong > Moderate > Limited > Disputed > Refuted.

DiseaseClassificationInheritance
SCN4A-related myopathy, autosomal recessiveDefinitiveAR

Mondo (26): hyperkalemic periodic paralysis (MONDO:0008224), paramyotonia congenita of Von Eulenburg (MONDO:0008195), hypokalemic periodic paralysis, type 2 (MONDO:0013234), congenital myasthenic syndrome 16 (MONDO:0013620), potassium-aggravated myotonia (MONDO:0018959), hypokalemic periodic paralysis, type 1 (MONDO:0042979), congenital myopathy 22A, classic (MONDO:0957247), congenital myopathy 22B, severe fetal (MONDO:0957265), muscular channelopathy (MONDO:0019119), long QT syndrome (MONDO:0002442), congenital myasthenic syndrome (MONDO:0018940), limb-girdle muscular dystrophy (MONDO:0016971), Batten-Turner congenital myopathy (MONDO:0100468), microcephaly (MONDO:0001149), myopathy (MONDO:0005336)

Orphanet (15): Hyperkalemic periodic paralysis (Orphanet:682), Congenital myasthenic syndrome (Orphanet:590), Potassium-aggravated myotonia (Orphanet:612), Hypokalemic periodic paralysis (Orphanet:681), Paramyotonia congenita of Von Eulenburg (Orphanet:684), Myotonia fluctuans (Orphanet:99734), Myotonia permanens (Orphanet:99735), Acetazolamide-responsive myotonia (Orphanet:99736), Muscular channelopathy (Orphanet:71864), Limb-girdle muscular dystrophy (Orphanet:263), Congenital myotonia (Orphanet:206973), Neuromuscular disease (Orphanet:68381), Arthrogryposis multiplex congenita (Orphanet:1037), Fetal akinesia deformation sequence (Orphanet:994), Skeletal muscle disease (Orphanet:98472)

HPO phenotypes

213 total (30 of 213 shown, HPO-id order):

HPOTerm
HP:0000006Autosomal dominant inheritance
HP:0000007Autosomal recessive inheritance
HP:0000194Open mouth
HP:0000207Triangular mouth
HP:0000218High palate
HP:0000276Long face
HP:0000278Retrognathia
HP:0000286Epicanthus
HP:0000322Short philtrum
HP:0000347Micrognathia
HP:0000369Low-set ears
HP:0000431Wide nasal bridge
HP:0000470Short neck
HP:0000486Strabismus
HP:0000490Deeply set eye
HP:0000494Downslanted palpebral fissures
HP:0000496Abnormality of eye movement
HP:0000508Ptosis
HP:0000520Proptosis
HP:0000544External ophthalmoplegia
HP:0000597Ophthalmoparesis
HP:0000602Ophthalmoplegia
HP:0000622Blurred vision
HP:0000651Diplopia
HP:0000664Synophrys
HP:0000678Dental crowding
HP:0000767Pectus excavatum
HP:0000771Gynecomastia
HP:0000821Hypothyroidism
HP:0000883Thin ribs

GWAS associations

7 associations (top):

StudyTraitp-value
GCST008163_374Height7.000000e-09
GCST008839_165Height3.000000e-16
GCST012227_346Hip circumference adjusted for BMI5.000000e-14
GCST012227_347Hip circumference adjusted for BMI2.000000e-13
GCST012227_348Hip circumference adjusted for BMI2.000000e-08
GCST012227_349Hip circumference adjusted for BMI2.000000e-08
GCST90020028_1586Hip circumference adjusted for BMI1.000000e-12

EFO canonical traits (1, from GWAS)

EFO IDTrait name
EFO:0008039BMI-adjusted hip circumference

MeSH disease descriptors (9)

DescriptorNameTree numbers
D020514Hypokalemic Periodic ParalysisC05.651.701.450; C10.668.491.650.450; C16.320.565.618.711.550; C18.452.648.618.711.550
D008133Long QT SyndromeC14.280.067.565; C14.280.123.625; C16.131.240.400.715; C23.550.073.547
D008831MicrocephalyC05.660.207.620; C10.500.507.400.500; C16.131.621.207.620; C16.131.666.507.400.500
D049288Muscular Dystrophies, Limb-GirdleC05.651.534.500.280; C10.668.491.175.500.149; C16.320.577.280
D020294Myasthenic Syndromes, CongenitalC10.668.758.800; C16.320.590
D009468Neuromuscular DiseasesC10.668
D020513Paralysis, Hyperkalemic PeriodicC05.651.701.600; C10.668.491.650.600; C16.320.565.618.711.600; C18.452.648.618.711.600
C567635Hypokalemic Periodic Paralysis, Type 2 (supp.)
C538353Potassium aggravated myotonia (supp.)

Drugs & pharmacology

Drug and pharmacology data

Is drug target: yes

ChEMBL targets (3): CHEMBL2072 (SINGLE PROTEIN), CHEMBL2331043 (PROTEIN FAMILY), CHEMBL4630762 (PROTEIN COMPLEX)

Molecules with ChEMBL bioactivity

24 molecules (phase ≥1), by development phase (incl. off-target/promiscuous compounds). Patent mentions across the top 20 by phase: 715,889 (via chembl_molecule»patent_compound — counts attach to the compound, not the gene–compound relationship, so off-target/promiscuous molecules can dominate).

MoleculeNamePhasePatents
CHEMBL108CARBAMAZEPINE453,528
CHEMBL16PHENYTOIN453,375
CHEMBL741LAMOTRIGINE428,962
CHEMBL744RILUZOLE420,055
CHEMBL79LIDOCAINE4140,673
CHEMBL11IMIPRAMINE448,893
CHEMBL12713SERTINDOLE48,984
CHEMBL1423PIMOZIDE417,310
CHEMBL193NIFEDIPINE474,353
CHEMBL23DILTIAZEM454,676
CHEMBL45816MIBEFRADIL47,838
CHEMBL54HALOPERIDOL460,883
CHEMBL558MEXILETINE415,463
CHEMBL629AMITRIPTYLINE452,595
CHEMBL633AMIODARONE429,704
CHEMBL71CHLORPROMAZINE445,827
CHEMBL3544913VIXOTRIGINE3374
CHEMBL3707392ELECLAZINE3111
CHEMBL507974TETRODOTOXIN31,034
CHEMBL113461TEDISAMIL31,251
CHEMBL475534NITRENDIPINE3
CHEMBL5314404AJMALINE3
CHEMBL2325014PF-050897712
CHEMBL87045CIFENLINE2

PharmGKB: 1 entry (VIP=true, CPIC=false)

PharmGKB clinical annotations

7 annotations.

VariantTypeLevelDrugsPhenotypes
rs121908547Efficacy3mexiletineMyotonic Disorders
rs121908547Efficacy3flecainideMyotonic Disorders
rs121908560Efficacy3mexiletineMyotonic Disorders
rs1567817380Efficacy3mexiletineMyotonic Disorders
rs1567817380Efficacy3flecainideMyotonic Disorders
rs80338792Efficacy3flecainide;mexiletineMyotonia
rs886043595Efficacy3mexiletineMyotonic Disorders

PharmGKB variants

6 variants.

VariantGenesLevelScore#Clin annotsDrugs
rs80338792SCN4A30.251flecainide;mexiletine
rs121908551SCN4A0.000
rs121908560SCN4A30.001mexiletine
rs886043595SCN4A30.001mexiletine
rs1567817380SCN4A30.002mexiletine;flecainide
rs121908547SCN4A30.002flecainide;mexiletine

GtoPdb / IUPHAR curated pharmacology

(IUPHAR/BPS Guide to Pharmacology — expert-curated)

Target class: vgic — Voltage-gated sodium channels (NaV)

Most potent curated ligand interactions (12 total), top 12:

LigandActionAffinityParameter
saxitoxinPore blocker8.4pIC50
tetrodotoxinPore blocker7.6pIC50
AFT-IISlows inactivation7.5pEC50
μ-conotoxin PIIIAAntagonist7.4pIC50
ATX-IISlows inactivation7.3pEC50
AM-6120Inhibition6.98pIC50
Bc-IIISlows inactivation6.1pEC50
GNE-616Inhibition6.0pKd
μ-conotoxin GIIIAPore blocker5.9pIC50
cannabidiolChannel blocker5.0pIC50
mexiletineAntagonist3.4pIC50
lidocainePore blocker2.7pIC50

Binding affinities (BindingDB)

572 measured of 576 human assays (576 total across all organisms); most potent 50 below. Values come from heterogeneous assays and are not directly comparable.

LigandMeasureValuePatent
5-cyclopropyl-4-[(3R)-1-[(3,5-dichlorophenyl)methyl]piperidin-3-yl]oxy-2-fluoro-N-methylsulfonylbenzamideIC500.3 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-N-cyclopropylsulfonyl-4-[[1-[(3,5-dichlorophenyl)-phenylmethyl]piperidin-4-yl]methoxy]-2-fluorobenzamideIC500.8 nMUS-9694002: Substituted benzamides and methods of use thereof
4-[(3R)-1-[[3-chloro-5-(trifluoromethoxy)phenyl]methyl]piperidin-3-yl]oxy-5-cyclopropyl-2-fluoro-N-methylsulfonylbenzamideIC501.3 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-4-[[1-[(3,5-dichlorophenyl)-phenylmethyl]piperidin-4-yl]methoxy]-2-fluoro-N-methylsulfonylbenzamideIC501.5 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-4-[[1-[(2S)-2-(3,5-dichlorophenyl)-1-methoxypropan-2-yl]piperidin-4-yl]methoxy]-2-fluoro-N-methylsulfonylbenzamideIC501.6 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-4-[[1-[(1S)-1-(3,5-dichlorophenyl)-2-methoxy-2-methylpropyl]piperidin-4-yl]methoxy]-N-ethylsulfonyl-2-fluorobenzamideIC501.6 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-4-[[1-[(1S)-1-(3,5-dichloro-2-fluorophenyl)ethyl]piperidin-4-yl]methoxy]-2-fluoro-N-methylsulfonylbenzamideIC501.6 nMUS-9694002: Substituted benzamides and methods of use thereof
4-[[1-[1-(2-chloro-4-fluorophenyl)-2,2,2-trifluoroethyl]piperidin-4-yl]methoxy]-5-cyclopropyl-2-fluoro-N-methylsulfonylbenzamideIC501.6 nMUS-9694002: Substituted benzamides and methods of use thereof
4-[(3R)-1-[(2-chlorophenyl)methyl]piperidin-3-yl]oxy-5-cyclopropyl-N-cyclopropylsulfonyl-2-fluorobenzamideIC501.7 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-4-[(3R)-1-[(2,4-dichlorophenyl)methyl]piperidin-3-yl]oxy-2-fluoro-N-methylsulfonylbenzamideIC501.7 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-4-[[1-[(1R)-1-(3,5-dichlorophenyl)-2-methoxy-2-methylpropyl]piperidin-4-yl]methoxy]-N-ethylsulfonyl-2-fluorobenzamideIC501.7 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-4-[[1-[2-(3,5-dichlorophenyl)propan-2-yl]piperidin-4-yl]methoxy]-2-fluoro-N-methylsulfonylbenzamideIC501.7 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-4-[[1-[(1S)-1-(3,5-dichlorophenyl)ethyl]-4-methylpiperidin-4-yl]methoxy]-2-fluoro-N-methylsulfonylbenzamideIC501.7 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-4-[[1-[(1S)-1-(3,5-dichlorophenyl)-2-methoxy-2-methylpropyl]piperidin-4-yl]methoxy]-2-fluoro-N-methylsulfonylbenzamideIC501.8 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-2-fluoro-4-[(3R)-1-[[4-fluoro-2-(trifluoromethyl)phenyl]methyl]piperidin-3-yl]oxy-N-methylsulfonylbenzamideIC501.9 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-4-[(3R)-1-[(1S)-1-(3,5-dichlorophenyl)ethyl]piperidin-3-yl]oxy-2-fluoro-N-methylsulfonylbenzamideIC501.9 nMUS-9694002: Substituted benzamides and methods of use thereof
4-[[1-[[3-chloro-5-(trifluoromethoxy)phenyl]methyl]piperidin-4-yl]methoxy]-5-cyclopropyl-2-fluoro-N-methylsulfonylbenzamideIC501.9 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-4-[[1-[2-(3,5-dichlorophenyl)propan-2-yl]piperidin-4-yl]methoxy]-N-ethylsulfonyl-2-fluorobenzamideIC501.9 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-4-[[1-[1-(3,5-dichlorophenyl)-2,2,2-trifluoroethyl]piperidin-4-yl]methoxy]-2-fluoro-N-methylsulfonylbenzamideIC501.9 nMUS-9694002: Substituted benzamides and methods of use thereof
N-(azetidin-1-ylsulfonyl)-4-[(3R,6R)-1-[(2-chloro-4-fluorophenyl)methyl]-6-methylpiperidin-3-yl]oxy-5-cyclopropyl-2-fluorobenzamideIC501.9 nMUS-9694002: Substituted benzamides and methods of use thereof
4-[(3R,6R)-1-[(2-chloro-4-fluorophenyl)methyl]-6-methylpiperidin-3-yl]oxy-5-cyclopropyl-N-cyclopropylsulfonyl-2-fluorobenzamideIC501.9 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-4-[[1-[1-(3,5-dichlorophenyl)propyl]piperidin-4-yl]methoxy]-2-fluoro-N-methylsulfonylbenzamideIC502 nMUS-9694002: Substituted benzamides and methods of use thereof
4-[[1-[bis(3-chlorophenyl)methyl]piperidin-4-yl]methoxy]-5-cyclopropyl-2-fluoro-N-methylsulfonylbenzamideIC502 nMUS-9694002: Substituted benzamides and methods of use thereof
4-[[1-[bis(2-chlorophenyl)methyl]piperidin-4-yl]methoxy]-5-cyclopropyl-2-fluoro-N-methylsulfonylbenzamideIC502 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-N-cyclopropylsulfonyl-4-[(3R)-1-(3,5-dichlorophenyl)piperidin-3-yl]oxy-2-fluorobenzamideIC502.1 nMUS-9694002: Substituted benzamides and methods of use thereof
4-[[1-[[2,5-bis(trifluoromethyl)phenyl]methyl]-4-methylpiperidin-4-yl]methoxy]-5-cyclopropyl-2-fluoro-N-methylsulfonylbenzamideIC502.1 nMUS-9694002: Substituted benzamides and methods of use thereof
4-[[1-[[3-chloro-5-(trifluoromethyl)phenyl]methyl]piperidin-4-yl]methoxy]-5-cyclopropyl-2-fluoro-N-methylsulfonylbenzamideIC502.1 nMUS-9694002: Substituted benzamides and methods of use thereof
4-[[1-[bis(4-chlorophenyl)methyl]piperidin-4-yl]methoxy]-5-cyclopropyl-N-cyclopropylsulfonyl-2-fluorobenzamideIC502.1 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-4-[(3R)-1-[(2,4-dimethylphenyl)methyl]piperidin-3-yl]oxy-2-fluoro-N-methylsulfonylbenzamideIC502.1 nMUS-9694002: Substituted benzamides and methods of use thereof
4-[[1-[5-chloro-4-(trifluoromethyl)-2-pyridinyl]-4-methylpiperidin-4-yl]methoxy]-5-cyclopropyl-2-fluoro-N-methylsulfonylbenzamideIC502.1 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-4-[[1-[(1S)-1-(2,4-dichlorophenyl)ethyl]piperidin-4-yl]methoxy]-2-fluoro-N-methylsulfonylbenzamideIC502.2 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-N-cyclopropylsulfonyl-4-[[1-[(3,5-dichlorophenyl)methyl]piperidin-4-yl]methoxy]-2-fluorobenzamideIC502.2 nMUS-9694002: Substituted benzamides and methods of use thereof
4-[(3R)-1-[(2-chloro-4-fluorophenyl)methyl]piperidin-3-yl]oxy-5-cyclopropyl-2-fluoro-N-methylsulfonylbenzamideIC502.2 nMUS-9694002: Substituted benzamides and methods of use thereof
4-[[(1R,5S)-3-[3-chloro-5-(trifluoromethoxy)phenoxy]-8-azabicyclo[3.2.1]octan-8-yl]methyl]-5-cyclopropyl-N-cyclopropylsulfonyl-2-fluorobenzamideIC502.2 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-4-[[4-(3,5-dichlorophenyl)sulfanylpiperidin-1-yl]methyl]-2-fluorobenzamideIC502.2 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-N-cyclopropylsulfonyl-4-[[1-[(1S)-1-(3,5-dichlorophenyl)-2-methoxyethyl]piperidin-4-yl]methoxy]-2-fluorobenzamideIC502.3 nMUS-9694002: Substituted benzamides and methods of use thereof
4-[(3R)-1-[(1S)-1-(2-chloro-4-fluorophenyl)ethyl]piperidin-3-yl]oxy-5-cyclopropyl-2-fluoro-N-methylsulfonylbenzamideIC502.3 nMUS-9694002: Substituted benzamides and methods of use thereof
4-[(3R)-1-[(1S)-1-(5-chloro-6-cyclopropyl-2-pyridinyl)ethyl]piperidin-3-yl]oxy-5-cyclopropyl-2-fluoro-N-methylsulfonylbenzamideIC502.3 nMUS-9694002: Substituted benzamides and methods of use thereof
4-[(3R)-1-[2-(3-chlorophenyl)propan-2-yl]piperidin-3-yl]oxy-5-cyclopropyl-2-fluorobenzamideIC502.3 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-N-cyclopropylsulfonyl-2-fluoro-4-[(3R)-1-[[4-fluoro-2-(trifluoromethyl)phenyl]methyl]piperidin-3-yl]oxybenzamideIC502.4 nMUS-9694002: Substituted benzamides and methods of use thereof
4-[[1-[[5-chloro-2-(trifluoromethyl)phenyl]methyl]-4-methylpiperidin-4-yl]methoxy]-5-cyclopropyl-2-fluoro-N-methylsulfonylbenzamideIC502.4 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-4-[[1-[(1R)-1-(3,5-dichlorophenyl)propyl]piperidin-4-yl]methoxy]-2-fluoro-N-methylsulfonylbenzamideIC502.4 nMUS-9694002: Substituted benzamides and methods of use thereof
N-(azetidin-1-ylsulfonyl)-4-[(3R,6S)-1-[(2-chloro-4-fluorophenyl)methyl]-6-methylpiperidin-3-yl]oxy-5-cyclopropyl-2-fluorobenzamideIC502.4 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-4-[[1-[(3,5-dichlorophenyl)-phenylmethyl]-3-methylazetidin-3-yl]methoxy]-2-fluoro-N-methylsulfonylbenzamideIC502.4 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-N-cyclopropylsulfonyl-4-[[1-[1-(3,5-dichlorophenyl)ethyl]piperidin-4-yl]methoxy]-2-fluorobenzamideIC502.5 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-4-[[1-[(1R)-1-(3,5-dichlorophenyl)-2-methoxy-2-methylpropyl]piperidin-4-yl]methoxy]-2-fluoro-N-methylsulfonylbenzamideIC502.5 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-4-[[1-[(2,4-dichlorophenyl)methyl]piperidin-4-yl]methoxy]-N-ethylsulfonyl-2-fluorobenzamideIC502.5 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-N-cyclopropylsulfonyl-4-[[1-[(3,5-dichlorophenyl)-phenylmethyl]-3-methylazetidin-3-yl]methoxy]-2-fluorobenzamideIC502.5 nMUS-9694002: Substituted benzamides and methods of use thereof
4-[(3R)-1-[(2-chloro-4-methylphenyl)methyl]piperidin-3-yl]oxy-5-cyclopropyl-2-fluoro-N-methylsulfonylbenzamideIC502.5 nMUS-9694002: Substituted benzamides and methods of use thereof
5-cyclopropyl-N-cyclopropylsulfonyl-4-[(3R)-1-[(3,5-dichlorophenyl)methyl]piperidin-3-yl]oxy-2-fluorobenzamideIC502.6 nMUS-9694002: Substituted benzamides and methods of use thereof

ChEMBL bioactivities

283 potent at pChembl≥5 of 350 total, top 50 by pChembl (potency: 10 = 0.1 nM, 6 = 1 µM).

pChemblTypeValueUnitMolecule
10.21IC500.062nMCHEMBL3617050
8.72IC501.9nMSAXITOXIN
8.70IC502nMCHEMBL5192731
8.70IC502nMCHEMBL5189924
8.47IC503.4nMCHEMBL5094715
8.12IC507.6nMTETRODOTOXIN
8.07IC508.5nMCHEMBL3657855
7.82IC5015nMCHEMBL5184022
7.72IC5019nMSAXITOXIN
7.70IC5020nMCHEMBL4524078
7.64IC5023nMCHEMBL4159473
7.64IC5023nMCHEMBL4170712
7.60IC5025nMTETRODOTOXIN
7.60Ki25nMCHEMBL38401
7.55Kd28nMCHEMBL4444707
7.52IC5030nMCHEMBL3318132
7.52IC5030nMSAXITOXIN
7.51Ki31nMCHEMBL37316
7.51Ki31nMCHEMBL289425
7.50IC5032nMCHEMBL5205795
7.48IC5033nMCHEMBL5192925
7.46Ki35nMCHEMBL37263
7.43IC5037nMCHEMBL4167379
7.41IC5039nMCHEMBL4160074
7.33Kd47nMCHEMBL4471012
7.29IC5051nMCHEMBL5204844
7.28IC5052nMCHEMBL4160943
7.28IC5053nMCHEMBL4169884
7.27IC5054nMPIMOZIDE
7.16IC5070nMCHEMBL3416931
7.16IC5070nMCHEMBL3798824
7.14IC5072nMCHEMBL4174312
7.12IC5075nMCHEMBL5204544
7.10IC5079nMCHEMBL3318132
7.05IC5090nMCHEMBL3416933
7.05IC5090nMSAXITOXIN
7.05IC5089nMCHEMBL4163637
7.04IC5092nMCHEMBL4162835
7.03IC5093nMCHEMBL3318147
7.00IC50100nMCHEMBL3416926
7.00IC50100nMCHEMBL3416930
7.00IC50100nMCHEMBL3983577
6.99IC50102nMCHEMBL4167720
6.98IC50104nMCHEMBL4159169
6.96IC50110nMCHEMBL3416937
6.96IC50110nMCHEMBL3416944
6.93IC50117nMCHEMBL4174592
6.92IC50120nMCHEMBL5208387
6.85IC50140nMCHEMBL3416934
6.85IC50140nMCHEMBL4588728

PubChem BioAssay actives

275 with measured affinity, of 602 total; 50 most potent distinct compounds. Largely complementary to BindingDB; screening values are coarse (µM, 4 dp), so sub-nM hits tie at the floor.

CompoundAssayTypeValueUnit
[(3R,5S,6S,11R,12S,16S)-14,17-diamino-19,19-dihydroxy-6-(hydroxymethyl)-10-oxo-3-sulfooxy-8-oxa-1,9,13,15,18-pentazapentacyclo[9.5.2.13,16.05,9.012,16]nonadeca-14,17-dien-13-yl]methyl N-hydroxycarbamate1246043: Inhibition of NaV1.4 channel (unknown origin) expressed in Xenopus laevis oocytes by two-electrode oocyte voltage-clamp methodic500.0001uM
[(3aS,4R,10aS)-2,6-diamino-10,10-dihydroxy-3a,4,8,9-tetrahydro-1H-pyrrolo[1,2-c]purin-4-yl]methyl carbamate1875797: Inhibition of human Nav1.4 expressed in HEK293 cells by whole cell patch clamp electrophysiology recordingic500.0019uM
N-[(3aS,4R,9S,10aS)-2,6-diamino-10,10-dihydroxy-4-(hydroxymethyl)-3a,4,8,9-tetrahydro-1H-pyrrolo[1,2-c]purin-9-yl]-2,5-bis(trifluoromethyl)benzamide1875797: Inhibition of human Nav1.4 expressed in HEK293 cells by whole cell patch clamp electrophysiology recordingic500.0020uM
N-[(3aS,4R,9S,10aS)-2,6-diamino-10,10-dihydroxy-4-(hydroxymethyl)-3a,4,8,9-tetrahydro-1H-pyrrolo[1,2-c]purin-9-yl]-2-(trifluoromethyl)benzamide1875797: Inhibition of human Nav1.4 expressed in HEK293 cells by whole cell patch clamp electrophysiology recordingic500.0020uM
5-cyclopropyl-4-[[1-[(1S)-1-(3,5-dichlorophenyl)ethyl]piperidin-4-yl]methoxy]-2-fluoro-N-methylsulfonylbenzamide1831649: Inhibition of full length human Nav1.4 expressed in HEK cells by whole cell voltage clamp analysisic500.0034uM
(1R,5R,6R,7R,9S,11S,12S,13S,14S)-3-amino-14-(hydroxymethyl)-8,10-dioxa-2,4-diazatetracyclo[7.3.1.17,11.01,6]tetradec-3-ene-5,9,12,13,14-pentol1525391: Inhibition of human Nav1.4 expressed in HEK293 cells by electrophysiology assayic500.0076uM
4-(1-adamantylmethoxy)-N-(azetidin-1-ylsulfonyl)-5-cyclopropyl-2-fluorobenzamide1831649: Inhibition of full length human Nav1.4 expressed in HEK cells by whole cell voltage clamp analysisic500.0085uM
N-[(3aS,4R,9S,10aS)-2,6-diamino-10,10-dihydroxy-4-(hydroxymethyl)-3a,4,8,9-tetrahydro-1H-pyrrolo[1,2-c]purin-9-yl]-2,3-dihydro-1H-indene-4-carboxamide1875797: Inhibition of human Nav1.4 expressed in HEK293 cells by whole cell patch clamp electrophysiology recordingic500.0150uM
(1R,4S,7S,10S,13S,16S,19S,22S,25S,28S,31S,34R,39R,42R,45S,52R)-39-amino-10-(4-aminobutyl)-45-(2-amino-2-oxoethyl)-19,31-bis(3-carbamimidamidopropyl)-22-(carboxymethyl)-4,7,28-tris(hydroxymethyl)-25-(1H-imidazol-4-ylmethyl)-13-(1H-indol-3-ylmethyl)-2,5,8,11,14,17,20,23,26,29,32,40,43,46,54-pentadecaoxo-36,37,49,50,56,57-hexathia-3,6,9,12,15,18,21,24,27,30,33,41,44,47,53-pentadecazatricyclo[32.13.7.416,42]octapentacontane-52-carboxylic acid1200071: Inhibition of human NaV1.4 at -140 mV holding potential by whole-cell patch clamp assayic500.0200uM
(4S)-4-[[(2S)-6-amino-2-[[(2S)-5-carbamimidamido-2-[[(1R,4S,7S,13S,16R,21R,24S,27S,30S,33S,36S,39S,42R,45S,48S,51S,54S,57S,60R,65R,68S,71S,74S)-27,51,74-tris(4-aminobutyl)-24-(3-amino-3-oxopropyl)-21-[[(2S)-2-[[(2S)-2-aminopent-4-ynoyl]amino]-3-(4-hydroxyphenyl)propanoyl]amino]-33-butyl-4,54-bis(3-carbamimidamidopropyl)-13-(2-carboxyethyl)-45-(carboxymethyl)-68-[(5-chloro-1H-indol-3-yl)methyl]-39-[(1R)-1-hydroxyethyl]-48-(hydroxymethyl)-30,36-bis(1H-indol-3-ylmethyl)-57-methyl-7,71-bis(2-methylpropyl)-3,6,9,12,15,22,25,28,31,34,37,40,43,46,49,52,55,58,67,70,73,76,81-tricosaoxo-18,19,62,63,78,79-hexathia-2,5,8,11,14,23,26,29,32,35,38,41,44,47,50,53,56,59,66,69,72,75,82-tricosazatricyclo[40.34.4.216,60]dooctacontane-65-carbonyl]amino]pentanoyl]amino]hexanoyl]amino]-5-[[(2S,3S)-1-amino-3-methyl-1-oxopentan-2-yl]amino]-5-oxopentanoic acid1363504: Inhibition of human Nav1.4 expressed in HEK293 cells at holding potential of -125 mV after 10 mins by patchxpress-based electrophysiology assayic500.0230uM
(4S)-4-[[(2S)-6-amino-2-[[(2S)-5-carbamimidamido-2-[[(1R,4S,7S,13S,16R,21R,24S,27S,30S,33S,36S,39S,42R,45S,48S,51S,54S,57S,60R,65R,68S,71S,74S)-27,51,74-tris(4-aminobutyl)-24-(3-amino-3-oxopropyl)-21-[[(2S)-2-[[(2S)-2-aminopent-4-ynoyl]amino]-3-(4-hydroxyphenyl)propanoyl]amino]-33-butyl-4,54-bis(3-carbamimidamidopropyl)-13-(2-carboxyethyl)-45-(carboxymethyl)-39-[(1R)-1-hydroxyethyl]-48-(hydroxymethyl)-30,36,68-tris(1H-indol-3-ylmethyl)-57-methyl-7,71-bis(2-methylpropyl)-3,6,9,12,15,22,25,28,31,34,37,40,43,46,49,52,55,58,67,70,73,76,81-tricosaoxo-18,19,62,63,78,79-hexathia-2,5,8,11,14,23,26,29,32,35,38,41,44,47,50,53,56,59,66,69,72,75,82-tricosazatricyclo[40.34.4.216,60]dooctacontane-65-carbonyl]amino]pentanoyl]amino]hexanoyl]amino]-5-[[(2S)-1-amino-1-oxo-4-phenylbutan-2-yl]amino]-5-oxopentanoic acid1363504: Inhibition of human Nav1.4 expressed in HEK293 cells at holding potential of -125 mV after 10 mins by patchxpress-based electrophysiology assayic500.0230uM
3-[4-(2,4-difluorophenoxy)phenyl]pyrazole-1-carboxamide205290: Affinity for inactive human SkM1 sodium channel expressed in HEK293 cellski0.0250uM
4-[[1-[[2-chloro-5-(trifluoromethyl)phenyl]methyl]piperidin-4-yl]methoxy]-5-cyclopropyl-2-fluoro-N-methylsulfonylbenzamide1525385: Binding affinity to human recombinant Nav1.4 by radioligand binding assaykd0.0280uM
(2S)-2-[[(2S)-2-[[(2S)-6-amino-2-[[(2S)-6-amino-2-[[(2S)-6-amino-2-[[(1R,4S,7S,13S,16R,21R,24S,27S,30S,33S,36S,39S,42R,45S,48S,51S,54S,57S,60R,65R,68S,71S,74S)-27,57-bis(4-aminobutyl)-21-[[(2S)-2-amino-3-(4-hydroxyphenyl)propanoyl]amino]-24-(3-amino-3-oxopropyl)-54,74-bis(3-carbamimidamidopropyl)-13,51-bis(2-carboxyethyl)-45-(carboxymethyl)-39-[(1R)-1-hydroxyethyl]-48-(hydroxymethyl)-30,36,68-tris(1H-indol-3-ylmethyl)-71-(2-methylpropyl)-7,33-bis(2-methylsulfanylethyl)-3,6,9,12,15,22,25,28,31,34,37,40,43,46,49,52,55,58,67,70,73,76,81-tricosaoxo-4-propan-2-yl-18,19,62,63,78,79-hexathia-2,5,8,11,14,23,26,29,32,35,38,41,44,47,50,53,56,59,66,69,72,75,82-tricosazatricyclo[40.34.4.216,60]dooctacontane-65-carbonyl]amino]hexanoyl]amino]hexanoyl]amino]hexanoyl]amino]-4-methylpentanoyl]amino]-3-(1H-indol-3-yl)propanoic acid1200071: Inhibition of human NaV1.4 at -140 mV holding potential by whole-cell patch clamp assayic500.0300uM
3-[4-(4-nitrophenoxy)phenyl]pyrazole-1-carboxamide205290: Affinity for inactive human SkM1 sodium channel expressed in HEK293 cellski0.0310uM
3-[4-(4-fluorophenoxy)phenyl]pyrazole-1-carboxamide205290: Affinity for inactive human SkM1 sodium channel expressed in HEK293 cellski0.0310uM
(3aS,4R,10aS)-2,6-diamino-4-(hydroxymethyl)-3a,4,8,9-tetrahydro-1H-pyrrolo[1,2-c]purine-10,10-diol1875797: Inhibition of human Nav1.4 expressed in HEK293 cells by whole cell patch clamp electrophysiology recordingic500.0320uM
[(3aS,4R,9S,10aS)-2,6-diamino-10,10-dihydroxy-4-(hydroxymethyl)-3a,4,8,9-tetrahydro-1H-pyrrolo[1,2-c]purin-9-yl] 2-chloro-4-fluorobenzoate1875797: Inhibition of human Nav1.4 expressed in HEK293 cells by whole cell patch clamp electrophysiology recordingic500.0330uM
3-(4-phenoxyphenyl)pyrazole-1-carboxamide205290: Affinity for inactive human SkM1 sodium channel expressed in HEK293 cellski0.0350uM
(4S)-4-[[(2S)-6-amino-2-[[(2S)-5-carbamimidamido-2-[[(1R,4S,7S,13S,16R,21R,24S,27S,30S,33S,36S,39S,42R,45S,48S,51S,54S,57S,60R,65R,68S,71S,74S)-27,51,74-tris(4-aminobutyl)-24-(3-amino-3-oxopropyl)-21-[[(2S)-2-[[(2S)-2-aminopent-4-ynoyl]amino]-3-(4-hydroxyphenyl)propanoyl]amino]-33-butyl-4,54-bis(3-carbamimidamidopropyl)-13-(2-carboxyethyl)-45-(carboxymethyl)-39-[(1R)-1-hydroxyethyl]-48-(hydroxymethyl)-30,36,68-tris(1H-indol-3-ylmethyl)-57-methyl-7,71-bis(2-methylpropyl)-3,6,9,12,15,22,25,28,31,34,37,40,43,46,49,52,55,58,67,70,73,76,81-tricosaoxo-18,19,62,63,78,79-hexathia-2,5,8,11,14,23,26,29,32,35,38,41,44,47,50,53,56,59,66,69,72,75,82-tricosazatricyclo[40.34.4.216,60]dooctacontane-65-carbonyl]amino]pentanoyl]amino]hexanoyl]amino]-5-[[(2S)-1-amino-3-cyclohexyl-1-oxopropan-2-yl]amino]-5-oxopentanoic acid1363504: Inhibition of human Nav1.4 expressed in HEK293 cells at holding potential of -125 mV after 10 mins by patchxpress-based electrophysiology assayic500.0370uM
(4S)-4-[[(2S)-6-amino-2-[[(2S)-5-carbamimidamido-2-[[(1R,4S,7S,13S,16R,21R,24S,27S,30S,33S,36S,39S,42R,45S,48S,51S,54S,57S,60R,65R,68S,71S,74S)-27,51,74-tris(4-aminobutyl)-24-(3-amino-3-oxopropyl)-21-[[(2S)-2-[[(2S)-2-aminopent-4-ynoyl]amino]-3-(4-hydroxyphenyl)propanoyl]amino]-68-[(6-bromo-1H-indol-3-yl)methyl]-33-butyl-4,54-bis(3-carbamimidamidopropyl)-13-(2-carboxyethyl)-45-(carboxymethyl)-39-[(1R)-1-hydroxyethyl]-48-(hydroxymethyl)-30,36-bis(1H-indol-3-ylmethyl)-57-methyl-7,71-bis(2-methylpropyl)-3,6,9,12,15,22,25,28,31,34,37,40,43,46,49,52,55,58,67,70,73,76,81-tricosaoxo-18,19,62,63,78,79-hexathia-2,5,8,11,14,23,26,29,32,35,38,41,44,47,50,53,56,59,66,69,72,75,82-tricosazatricyclo[40.34.4.216,60]dooctacontane-65-carbonyl]amino]pentanoyl]amino]hexanoyl]amino]-5-[[(2S,3S)-1-amino-3-methyl-1-oxopentan-2-yl]amino]-5-oxopentanoic acid1363504: Inhibition of human Nav1.4 expressed in HEK293 cells at holding potential of -125 mV after 10 mins by patchxpress-based electrophysiology assayic500.0390uM
N-cyclopropylsulfonyl-1-[(3R)-1-[(1R)-1-(3,5-dichlorophenyl)ethyl]pyrrolidin-3-yl]-6-fluoro-3-methylindazole-5-carboxamide1525385: Binding affinity to human recombinant Nav1.4 by radioligand binding assaykd0.0470uM
[(3aS,4R,9S,10aS)-2,6-diamino-10,10-dihydroxy-4-(hydroxymethyl)-3a,4,8,9-tetrahydro-1H-pyrrolo[1,2-c]purin-9-yl] 2-(trifluoromethyl)benzoate1875797: Inhibition of human Nav1.4 expressed in HEK293 cells by whole cell patch clamp electrophysiology recordingic500.0510uM
(4S)-4-[[(2S)-6-amino-2-[[(2S)-5-carbamimidamido-2-[[(1R,4S,7S,13S,16R,21R,24S,27S,30S,33S,36S,39S,42R,45S,48S,51S,54S,57S,60R,65R,68S,71S,74S)-27,51,74-tris(4-aminobutyl)-24-(3-amino-3-oxopropyl)-21-[[(2S)-2-[[(2S)-2-aminopent-4-ynoyl]amino]-3-(4-hydroxyphenyl)propanoyl]amino]-30-benzyl-33-butyl-4,54-bis(3-carbamimidamidopropyl)-13-(2-carboxyethyl)-45-(carboxymethyl)-39-[(1R)-1-hydroxyethyl]-48-(hydroxymethyl)-36,68-bis(1H-indol-3-ylmethyl)-57-methyl-7,71-bis(2-methylpropyl)-3,6,9,12,15,22,25,28,31,34,37,40,43,46,49,52,55,58,67,70,73,76,81-tricosaoxo-18,19,62,63,78,79-hexathia-2,5,8,11,14,23,26,29,32,35,38,41,44,47,50,53,56,59,66,69,72,75,82-tricosazatricyclo[40.34.4.216,60]dooctacontane-65-carbonyl]amino]pentanoyl]amino]hexanoyl]amino]-5-[[(2S,3S)-1-amino-3-methyl-1-oxopentan-2-yl]amino]-5-oxopentanoic acid1363504: Inhibition of human Nav1.4 expressed in HEK293 cells at holding potential of -125 mV after 10 mins by patchxpress-based electrophysiology assayic500.0520uM
(4S)-4-[[(2S)-6-amino-2-[[(2S)-5-carbamimidamido-2-[[(1R,4S,7S,13S,16R,21R,24S,27S,30S,33S,36S,39S,42R,45S,48S,51S,54S,57S,60R,65R,68S,71S,74S)-27,51,74-tris(4-aminobutyl)-24-(3-amino-3-oxopropyl)-21-[[(2S)-2-[[(2S)-2-aminopent-4-ynoyl]amino]-3-(4-hydroxyphenyl)propanoyl]amino]-68-[(5-bromo-1H-indol-3-yl)methyl]-33-butyl-4,54-bis(3-carbamimidamidopropyl)-13-(2-carboxyethyl)-45-(carboxymethyl)-39-[(1R)-1-hydroxyethyl]-48-(hydroxymethyl)-30,36-bis(1H-indol-3-ylmethyl)-57-methyl-7,71-bis(2-methylpropyl)-3,6,9,12,15,22,25,28,31,34,37,40,43,46,49,52,55,58,67,70,73,76,81-tricosaoxo-18,19,62,63,78,79-hexathia-2,5,8,11,14,23,26,29,32,35,38,41,44,47,50,53,56,59,66,69,72,75,82-tricosazatricyclo[40.34.4.216,60]dooctacontane-65-carbonyl]amino]pentanoyl]amino]hexanoyl]amino]-5-[[(2S,3S)-1-amino-3-methyl-1-oxopentan-2-yl]amino]-5-oxopentanoic acid1363504: Inhibition of human Nav1.4 expressed in HEK293 cells at holding potential of -125 mV after 10 mins by patchxpress-based electrophysiology assayic500.0530uM
Pimozide1207165: Inhibition of Na channel (species unknown)ic500.0540uM
(3S)-3-amino-4-[[(1R,4S,10S,13S,16S,19S,22S,25R,30R,33S,36S,39S,42S,45S,48S,51R,54S,57S,60S,63S,69S,72R,77R,80S,86S,89S,92S,95S)-30-[[(2S)-6-amino-1-[[(2S)-1-[[(2S)-1-[[(2S)-1-amino-1-oxo-3-phenylpropan-2-yl]amino]-3-methyl-1-oxobutan-2-yl]amino]-3-(4-hydroxyphenyl)-1-oxopropan-2-yl]amino]-1-oxohexan-2-yl]carbamoyl]-22,36,95-tris(4-aminobutyl)-16,60-bis(2-amino-2-oxoethyl)-86-benzyl-4-[(2S)-butan-2-yl]-89-butyl-45,69,92-tris(3-carbamimidamidopropyl)-13,19-bis(carboxymethyl)-42-[(1R)-1-hydroxyethyl]-48-(hydroxymethyl)-39-(1H-imidazol-5-ylmethyl)-33-(1H-indol-3-ylmethyl)-57,80-bis(2-methylpropyl)-2,3a,5,11,14,17,20,23,32,35,38,41,44,47,50,53,56,59,62,68,71,78,81,84,87,90,93,96-octacosaoxo-54-propan-2-yl-a,27,28,74,75,99-hexathia-2a,3,6,12,15,18,21,24,31,34,37,40,43,46,49,52,55,58,61,67,70,79,82,85,88,91,94,97-octacosazapentacyclo[49.46.4.225,72.06,10.063,67]trihectan-77-yl]amino]-4-oxobutanoic acid1200071: Inhibition of human NaV1.4 at -140 mV holding potential by whole-cell patch clamp assayic500.0700uM
(3S)-3-amino-4-[[(1R,4S,10S,13S,16S,19S,22S,25R,30R,33S,36S,39S,42S,45S,48S,51R,54S,57S,60S,63S,69S,72R,77R,80S,86S,89S,92S,95S)-30-[[(2S)-6-amino-1-[[(2S)-1-[[(2S)-1-[[(2S)-1-amino-1-oxo-3-phenylpropan-2-yl]amino]-3-methyl-1-oxobutan-2-yl]amino]-3-naphthalen-1-yl-1-oxopropan-2-yl]amino]-1-oxohexan-2-yl]carbamoyl]-22,36,95-tris(4-aminobutyl)-16,60-bis(2-amino-2-oxoethyl)-86-benzyl-4-[(2S)-butan-2-yl]-45,69,92-tris(3-carbamimidamidopropyl)-13,19-bis(carboxymethyl)-42-[(1R)-1-hydroxyethyl]-48-(hydroxymethyl)-39-(1H-imidazol-5-ylmethyl)-33-(1H-indol-3-ylmethyl)-57,80-bis(2-methylpropyl)-89-(2-methylsulfanylethyl)-2,3a,5,11,14,17,20,23,32,35,38,41,44,47,50,53,56,59,62,68,71,78,81,84,87,90,93,96-octacosaoxo-54-propan-2-yl-a,27,28,74,75,99-hexathia-2a,3,6,12,15,18,21,24,31,34,37,40,43,46,49,52,55,58,61,67,70,79,82,85,88,91,94,97-octacosazapentacyclo[49.46.4.225,72.06,10.063,67]trihectan-77-yl]amino]-4-oxobutanoic acid1296072: Inhibition of human Nav1.4 expressed in HEK293 cells by whole cell patch clamp electrophysiology methodic500.0700uM
(4S)-4-amino-5-[[(2S)-1-[[(1R,4S,7S,13S,16R,21R,24S,27S,30S,33S,36S,39S,42R,45S,48S,51S,54S,57S,60R,65R,68S,71S,74S)-65-[[(2S)-1-[[(2S)-6-amino-1-[[(2S)-1-[[(2S,3S)-1-amino-3-methyl-1-oxopentan-2-yl]amino]-4-carboxy-1-oxobutan-2-yl]amino]-1-oxohexan-2-yl]amino]-5-carbamimidamido-1-oxopentan-2-yl]carbamoyl]-27,74-bis(4-aminobutyl)-24-(3-amino-3-oxopropyl)-68-[(5-bromo-1H-indol-3-yl)methyl]-33-butyl-4,54-bis(3-carbamimidamidopropyl)-13,51-bis(2-carboxyethyl)-45-(carboxymethyl)-39-[(1R)-1-hydroxyethyl]-48-(hydroxymethyl)-30,36-bis(1H-indol-3-ylmethyl)-57-methyl-7,71-bis(2-methylpropyl)-3,6,9,12,15,22,25,28,31,34,37,40,43,46,49,52,55,58,67,70,73,76,81-tricosaoxo-18,19,62,63,78,79-hexathia-2,5,8,11,14,23,26,29,32,35,38,41,44,47,50,53,56,59,66,69,72,75,82-tricosazatricyclo[40.34.4.216,60]dooctacontan-21-yl]amino]-1-oxopent-4-yn-2-yl]amino]-5-oxopentanoic acid1363504: Inhibition of human Nav1.4 expressed in HEK293 cells at holding potential of -125 mV after 10 mins by patchxpress-based electrophysiology assayic500.0720uM
N-[(3aS,4R,9S,10aS)-2,6-diamino-10,10-dihydroxy-4-(hydroxymethyl)-3a,4,8,9-tetrahydro-1H-pyrrolo[1,2-c]purin-9-yl]-2,4-bis(trifluoromethyl)benzamide1875797: Inhibition of human Nav1.4 expressed in HEK293 cells by whole cell patch clamp electrophysiology recordingic500.0750uM
(4S)-4-amino-5-[[(2S)-1-[[(1R,4S,7S,13S,16R,21R,24S,27S,30S,33S,36S,39S,42R,45S,48S,51S,54S,57S,60R,65R,68S,71S,74S)-65-[[(2S)-1-[[(2S)-6-amino-1-[[(2S)-1-[[(2S,3S)-1-amino-3-methyl-1-oxopentan-2-yl]amino]-4-carboxy-1-oxobutan-2-yl]amino]-1-oxohexan-2-yl]amino]-5-carbamimidamido-1-oxopentan-2-yl]carbamoyl]-27,51,74-tris(4-aminobutyl)-24-(3-amino-3-oxopropyl)-68-[(5-bromo-1H-indol-3-yl)methyl]-33-butyl-4,54-bis(3-carbamimidamidopropyl)-13,48-bis(2-carboxyethyl)-45-(carboxymethyl)-39-[(1R)-1-hydroxyethyl]-30,36-bis(1H-indol-3-ylmethyl)-57-methyl-7,71-bis(2-methylpropyl)-3,6,9,12,15,22,25,28,31,34,37,40,43,46,49,52,55,58,67,70,73,76,81-tricosaoxo-18,19,62,63,78,79-hexathia-2,5,8,11,14,23,26,29,32,35,38,41,44,47,50,53,56,59,66,69,72,75,82-tricosazatricyclo[40.34.4.216,60]dooctacontan-21-yl]amino]-1-oxopent-4-yn-2-yl]amino]-5-oxopentanoic acid1363504: Inhibition of human Nav1.4 expressed in HEK293 cells at holding potential of -125 mV after 10 mins by patchxpress-based electrophysiology assayic500.0890uM
(3S)-3-amino-4-[[(1R,4S,10S,13S,16S,19S,22S,25R,30R,33S,36S,39S,42S,45S,48S,51R,54S,57S,60S,63S,69S,72R,77R,80S,86S,89S,92S,95S)-30-[[(2S)-6-amino-1-[[(2S)-1-[[(2S)-1-[[(2S)-1-amino-1-oxo-3-phenylpropan-2-yl]amino]-3-methyl-1-oxobutan-2-yl]amino]-3-(4-hydroxyphenyl)-1-oxopropan-2-yl]amino]-1-oxohexan-2-yl]carbamoyl]-22,36,95-tris(4-aminobutyl)-16,60-bis(2-amino-2-oxoethyl)-86-benzyl-4-[(2S)-butan-2-yl]-45,69,92-tris(3-carbamimidamidopropyl)-13,19-bis(carboxymethyl)-89-(cyclohexylmethyl)-42-[(1R)-1-hydroxyethyl]-48-(hydroxymethyl)-39-(1H-imidazol-5-ylmethyl)-33-(1H-indol-3-ylmethyl)-57,80-bis(2-methylpropyl)-2,3a,5,11,14,17,20,23,32,35,38,41,44,47,50,53,56,59,62,68,71,78,81,84,87,90,93,96-octacosaoxo-54-propan-2-yl-a,27,28,74,75,99-hexathia-2a,3,6,12,15,18,21,24,31,34,37,40,43,46,49,52,55,58,61,67,70,79,82,85,88,91,94,97-octacosazapentacyclo[49.46.4.225,72.06,10.063,67]trihectan-77-yl]amino]-4-oxobutanoic acid1200071: Inhibition of human NaV1.4 at -140 mV holding potential by whole-cell patch clamp assayic500.0900uM
(4S)-4-[[(2S)-6-amino-2-[[(2S)-5-carbamimidamido-2-[[(1R,4S,7S,13S,16R,21R,24S,27S,30S,33S,36S,39S,42R,45S,48S,51S,54S,57S,60R,65R,68S,71S,74S)-27,51,74-tris(4-aminobutyl)-24-(3-amino-3-oxopropyl)-21-[[(2S)-2-[[(2S)-2-aminopent-4-ynoyl]amino]-3-(4-hydroxyphenyl)propanoyl]amino]-33-butyl-4,54-bis(3-carbamimidamidopropyl)-13-(2-carboxyethyl)-45-(carboxymethyl)-39-[(1R)-1-hydroxyethyl]-48-(hydroxymethyl)-30,36,68-tris(1H-indol-3-ylmethyl)-57-methyl-7,71-bis(2-methylpropyl)-3,6,9,12,15,22,25,28,31,34,37,40,43,46,49,52,55,58,67,70,73,76,81-tricosaoxo-18,19,62,63,78,79-hexathia-2,5,8,11,14,23,26,29,32,35,38,41,44,47,50,53,56,59,66,69,72,75,82-tricosazatricyclo[40.34.4.216,60]dooctacontane-65-carbonyl]amino]pentanoyl]amino]hexanoyl]amino]-5-[[(2S)-1-amino-3-(1H-indol-3-yl)-1-oxopropan-2-yl]amino]-5-oxopentanoic acid1363504: Inhibition of human Nav1.4 expressed in HEK293 cells at holding potential of -125 mV after 10 mins by patchxpress-based electrophysiology assayic500.0920uM
3-[(1R,4S,7S,13S,16R,21R,24S,27S,30S,33S,36S,39S,42R,45S,48S,51S,54S,57S,60R,65R,68S,71S,74S)-65-[[(2S)-6-amino-1-[[(2S)-6-amino-1-[[(2S)-6-amino-1-[[(2S)-1-[[(2S)-1-amino-3-(1H-indol-3-yl)-1-oxopropan-2-yl]amino]-4-methyl-1-oxopentan-2-yl]amino]-1-oxohexan-2-yl]amino]-1-oxohexan-2-yl]amino]-1-oxohexan-2-yl]carbamoyl]-27,57-bis(4-aminobutyl)-21-[[(2S)-2-amino-3-(4-hydroxyphenyl)propanoyl]amino]-24-(3-amino-3-oxopropyl)-54,74-bis(3-carbamimidamidopropyl)-51-(2-carboxyethyl)-45-(carboxymethyl)-39-[(1R)-1-hydroxyethyl]-48-(hydroxymethyl)-30,36,68-tris(1H-indol-3-ylmethyl)-71-(2-methylpropyl)-7,33-bis(2-methylsulfanylethyl)-3,6,9,12,15,22,25,28,31,34,37,40,43,46,49,52,55,58,67,70,73,76,81-tricosaoxo-4-propan-2-yl-18,19,62,63,78,79-hexathia-2,5,8,11,14,23,26,29,32,35,38,41,44,47,50,53,56,59,66,69,72,75,82-tricosazatricyclo[40.34.4.216,60]dooctacontan-13-yl]propanoic acid1818596: Inhibition of human Nav1.4 stably expressed in cells with train pulses to -20 mV at 0.1 Hz and holding potential of -95 mV by whole-cell Qube-automated patch clamp assayic500.0930uM
(3S)-3-amino-4-[[(1R,4S,10S,13S,16S,19S,22S,25R,30R,33S,36S,39S,42S,45S,48S,51R,54S,57S,60S,63S,69S,72R,77R,80S,86S,89S,92S,95S)-30-[[(2S)-6-amino-1-[[(2S)-1-[[(2S)-1-[[(2S)-1-amino-1-oxo-3-phenylpropan-2-yl]amino]-3-methyl-1-oxobutan-2-yl]amino]-3-(4-hydroxyphenyl)-1-oxopropan-2-yl]amino]-1-oxohexan-2-yl]carbamoyl]-22,36,95-tris(4-aminobutyl)-16,60-bis(2-amino-2-oxoethyl)-4-[(2S)-butan-2-yl]-45,69,92-tris(3-carbamimidamidopropyl)-13,19-bis(carboxymethyl)-86-(cyclohexylmethyl)-42-[(1R)-1-hydroxyethyl]-48-(hydroxymethyl)-39-(1H-imidazol-5-ylmethyl)-33-(1H-indol-3-ylmethyl)-57,80-bis(2-methylpropyl)-89-(2-methylsulfanylethyl)-2,3a,5,11,14,17,20,23,32,35,38,41,44,47,50,53,56,59,62,68,71,78,81,84,87,90,93,96-octacosaoxo-54-propan-2-yl-a,27,28,74,75,99-hexathia-2a,3,6,12,15,18,21,24,31,34,37,40,43,46,49,52,55,58,61,67,70,79,82,85,88,91,94,97-octacosazapentacyclo[49.46.4.225,72.06,10.063,67]trihectan-77-yl]amino]-4-oxobutanoic acid1200071: Inhibition of human NaV1.4 at -140 mV holding potential by whole-cell patch clamp assayic500.1000uM
(3S)-3-amino-4-[[(1R,4S,10S,13S,16S,19S,22S,25R,30R,33S,36S,39S,42S,45S,48S,51R,54S,57S,60S,63S,69S,72R,77R,80S,86S,89S,92S,95S)-30-[[(2S)-6-amino-1-[[(2S)-1-[[(2S)-1-[[(2S)-1-amino-1-oxo-3-phenylpropan-2-yl]amino]-3-methyl-1-oxobutan-2-yl]amino]-3-(4-hydroxyphenyl)-1-oxopropan-2-yl]amino]-1-oxohexan-2-yl]carbamoyl]-22,36,95-tris(4-aminobutyl)-16,60-bis(2-amino-2-oxoethyl)-4-[(2S)-butan-2-yl]-45,69,92-tris(3-carbamimidamidopropyl)-13,19-bis(carboxymethyl)-42-[(1R)-1-hydroxyethyl]-48-(hydroxymethyl)-39-(1H-imidazol-5-ylmethyl)-33-(1H-indol-3-ylmethyl)-57,80-bis(2-methylpropyl)-89-(2-methylsulfanylethyl)-2,3a,5,11,14,17,20,23,32,35,38,41,44,47,50,53,56,59,62,68,71,78,81,84,87,90,93,96-octacosaoxo-86-[(4-phenylphenyl)methyl]-54-propan-2-yl-a,27,28,74,75,99-hexathia-2a,3,6,12,15,18,21,24,31,34,37,40,43,46,49,52,55,58,61,67,70,79,82,85,88,91,94,97-octacosazapentacyclo[49.46.4.225,72.06,10.063,67]trihectan-77-yl]amino]-4-oxobutanoic acid1200071: Inhibition of human NaV1.4 at -140 mV holding potential by whole-cell patch clamp assayic500.1000uM
(4S)-4-[[(2S)-6-amino-2-[[(2S)-5-carbamimidamido-2-[[(1R,4S,7S,13S,16R,21R,24S,27S,30S,33S,36S,39S,42R,45S,48S,51S,54S,57S,60R,65R,68S,71S,74S)-27,51,74-tris(4-aminobutyl)-24-(3-amino-3-oxopropyl)-21-[[(2S)-2-[[(2S)-2-aminopent-4-ynoyl]amino]-3-(4-hydroxyphenyl)propanoyl]amino]-33,71-dibutyl-4,54-bis(3-carbamimidamidopropyl)-13-(2-carboxyethyl)-45-(carboxymethyl)-39-[(1R)-1-hydroxyethyl]-48-(hydroxymethyl)-30,36,68-tris(1H-indol-3-ylmethyl)-57-methyl-7-(2-methylpropyl)-3,6,9,12,15,22,25,28,31,34,37,40,43,46,49,52,55,58,67,70,73,76,81-tricosaoxo-18,19,62,63,78,79-hexathia-2,5,8,11,14,23,26,29,32,35,38,41,44,47,50,53,56,59,66,69,72,75,82-tricosazatricyclo[40.34.4.216,60]dooctacontane-65-carbonyl]amino]pentanoyl]amino]hexanoyl]amino]-5-[[(2S,3S)-1-amino-3-methyl-1-oxopentan-2-yl]amino]-5-oxopentanoic acid1363504: Inhibition of human Nav1.4 expressed in HEK293 cells at holding potential of -125 mV after 10 mins by patchxpress-based electrophysiology assayic500.1020uM
(4S)-4-amino-5-[[(2S)-1-[[(1R,4S,7S,13S,16R,21R,24S,27S,30S,33S,36S,39S,42R,45S,48S,51S,54S,57S,60R,65R,68S,71S,74S)-65-[[(2S)-1-[[(2S)-6-amino-1-[[(2S)-1-[[(2S,3S)-1-amino-3-methyl-1-oxopentan-2-yl]amino]-4-carboxy-1-oxobutan-2-yl]amino]-1-oxohexan-2-yl]amino]-5-carbamimidamido-1-oxopentan-2-yl]carbamoyl]-27,74-bis(4-aminobutyl)-24-(3-amino-3-oxopropyl)-68-[(5-bromo-1H-indol-3-yl)methyl]-33-butyl-4,54-bis(3-carbamimidamidopropyl)-13-(2-carboxyethyl)-45-(carboxymethyl)-39-[(1R)-1-hydroxyethyl]-48-(hydroxymethyl)-30,36-bis(1H-indol-3-ylmethyl)-51,57-dimethyl-7,71-bis(2-methylpropyl)-3,6,9,12,15,22,25,28,31,34,37,40,43,46,49,52,55,58,67,70,73,76,81-tricosaoxo-18,19,62,63,78,79-hexathia-2,5,8,11,14,23,26,29,32,35,38,41,44,47,50,53,56,59,66,69,72,75,82-tricosazatricyclo[40.34.4.216,60]dooctacontan-21-yl]amino]-1-oxopent-4-yn-2-yl]amino]-5-oxopentanoic acid1363504: Inhibition of human Nav1.4 expressed in HEK293 cells at holding potential of -125 mV after 10 mins by patchxpress-based electrophysiology assayic500.1040uM
(3S)-3-amino-4-[[(1R,4S,10S,13S,16S,19S,22S,25R,30R,33S,36S,39S,42S,45S,48S,51R,54S,57S,60S,63S,69S,72R,77R,80S,86S,89S,92S,95S)-30-[[(2S)-6-amino-1-[[(2S)-1-[[(2S)-1-[[(2S)-1-amino-1-oxo-3-phenylpropan-2-yl]amino]-3-methyl-1-oxobutan-2-yl]amino]-3-(4-hydroxyphenyl)-1-oxopropan-2-yl]amino]-1-oxohexan-2-yl]carbamoyl]-22,36,95-tris(4-aminobutyl)-16,60-bis(2-amino-2-oxoethyl)-86-benzyl-4-[(2S)-butan-2-yl]-45,69,92-tris(3-carbamimidamidopropyl)-13,19-bis(carboxymethyl)-42-[(1R)-1-hydroxyethyl]-48-(hydroxymethyl)-39-(1H-imidazol-5-ylmethyl)-33-(1H-indol-3-ylmethyl)-57,80-bis(2-methylpropyl)-89-(naphthalen-1-ylmethyl)-2,3a,5,11,14,17,20,23,32,35,38,41,44,47,50,53,56,59,62,68,71,78,81,84,87,90,93,96-octacosaoxo-54-propan-2-yl-a,27,28,74,75,99-hexathia-2a,3,6,12,15,18,21,24,31,34,37,40,43,46,49,52,55,58,61,67,70,79,82,85,88,91,94,97-octacosazapentacyclo[49.46.4.225,72.06,10.063,67]trihectan-77-yl]amino]-4-oxobutanoic acid1200071: Inhibition of human NaV1.4 at -140 mV holding potential by whole-cell patch clamp assayic500.1100uM
(3S)-3-amino-4-[[(1R,4S,10S,13S,16S,19S,22S,25R,30R,33S,36S,39S,42S,45S,48S,51R,54S,57S,60S,63S,69S,72R,77R,80S,86S,89S,92S,95S)-30-[[(2S)-6-amino-1-[[(2S)-1-[[(2S)-1-[[(2S)-1-amino-1-oxo-3-phenylpropan-2-yl]amino]-3-methyl-1-oxobutan-2-yl]amino]-3-(4-hydroxyphenyl)-1-oxopropan-2-yl]amino]-1-oxohexan-2-yl]carbamoyl]-22,36,95-tris(4-aminobutyl)-16,60-bis(2-amino-2-oxoethyl)-86-benzyl-4-[(2S)-butan-2-yl]-42,45,69,92-tetrakis(3-carbamimidamidopropyl)-13,19-bis(carboxymethyl)-48-(hydroxymethyl)-39-(1H-imidazol-5-ylmethyl)-33-(1H-indol-3-ylmethyl)-57,80-bis(2-methylpropyl)-89-(2-methylsulfanylethyl)-2,3a,5,11,14,17,20,23,32,35,38,41,44,47,50,53,56,59,62,68,71,78,81,84,87,90,93,96-octacosaoxo-54-propan-2-yl-a,27,28,74,75,99-hexathia-2a,3,6,12,15,18,21,24,31,34,37,40,43,46,49,52,55,58,61,67,70,79,82,85,88,91,94,97-octacosazapentacyclo[49.46.4.225,72.06,10.063,67]trihectan-77-yl]amino]-4-oxobutanoic acid1200071: Inhibition of human NaV1.4 at -140 mV holding potential by whole-cell patch clamp assayic500.1100uM
(4S)-4-[[(2S)-6-amino-2-[[(2S)-5-carbamimidamido-2-[[(1R,4S,7S,13S,16R,21R,24S,27S,30S,33S,36S,39S,42R,45S,48S,51S,54S,57S,60R,65R,68S,71S,74S)-27,51,74-tris(4-aminobutyl)-24-(3-amino-3-oxopropyl)-21-[[(2S)-2-[[(2S)-2-aminopent-4-ynoyl]amino]-3-(4-hydroxyphenyl)propanoyl]amino]-33-butyl-4,54-bis(3-carbamimidamidopropyl)-13-(2-carboxyethyl)-45-(carboxymethyl)-71-(cyclohexylmethyl)-39-[(1R)-1-hydroxyethyl]-48-(hydroxymethyl)-30,36,68-tris(1H-indol-3-ylmethyl)-57-methyl-7-(2-methylpropyl)-3,6,9,12,15,22,25,28,31,34,37,40,43,46,49,52,55,58,67,70,73,76,81-tricosaoxo-18,19,62,63,78,79-hexathia-2,5,8,11,14,23,26,29,32,35,38,41,44,47,50,53,56,59,66,69,72,75,82-tricosazatricyclo[40.34.4.216,60]dooctacontane-65-carbonyl]amino]pentanoyl]amino]hexanoyl]amino]-5-[[(2S,3S)-1-amino-3-methyl-1-oxopentan-2-yl]amino]-5-oxopentanoic acid1363504: Inhibition of human Nav1.4 expressed in HEK293 cells at holding potential of -125 mV after 10 mins by patchxpress-based electrophysiology assayic500.1170uM
N-[(3aS,4R,9S,10aS)-2,6-diamino-10,10-dihydroxy-4-(hydroxymethyl)-3a,4,8,9-tetrahydro-1H-pyrrolo[1,2-c]purin-9-yl]-3,3-dimethyl-2H-1-benzofuran-7-carboxamide1875797: Inhibition of human Nav1.4 expressed in HEK293 cells by whole cell patch clamp electrophysiology recordingic500.1200uM
(3S)-3-amino-4-[[(1R,4S,10S,13S,16S,19S,22S,25R,30R,33S,36S,39S,42S,45S,48S,51R,54S,57S,60S,63S,69S,72R,77R,80S,86S,89S,92S,95S)-30-[[(2S)-6-amino-1-[[(2S)-1-[[(2S)-1-[[(2S)-1-amino-1-oxo-3-phenylpropan-2-yl]amino]-3-methyl-1-oxobutan-2-yl]amino]-3-(4-hydroxyphenyl)-1-oxopropan-2-yl]amino]-1-oxohexan-2-yl]carbamoyl]-22,36,95-tris(4-aminobutyl)-16,60-bis(2-amino-2-oxoethyl)-86,89-dibenzyl-4-[(2S)-butan-2-yl]-45,69,92-tris(3-carbamimidamidopropyl)-13,19-bis(carboxymethyl)-42-[(1R)-1-hydroxyethyl]-48-(hydroxymethyl)-39-(1H-imidazol-5-ylmethyl)-33-(1H-indol-3-ylmethyl)-57,80-bis(2-methylpropyl)-2,3a,5,11,14,17,20,23,32,35,38,41,44,47,50,53,56,59,62,68,71,78,81,84,87,90,93,96-octacosaoxo-54-propan-2-yl-a,27,28,74,75,99-hexathia-2a,3,6,12,15,18,21,24,31,34,37,40,43,46,49,52,55,58,61,67,70,79,82,85,88,91,94,97-octacosazapentacyclo[49.46.4.225,72.06,10.063,67]trihectan-77-yl]amino]-4-oxobutanoic acid1200071: Inhibition of human NaV1.4 at -140 mV holding potential by whole-cell patch clamp assayic500.1400uM
(3S)-3-amino-4-oxo-4-[[(1R,4S,7S,10S,13S,16S,19R,24R,27S,30S,33S,36S,39S,42S,45R,51S,54S,57S,60R,65R,68S,71S,74S,77S,83S)-7,30,36,42,57-pentakis(4-aminobutyl)-77-benzyl-39,68-bis(3-carbamimidamidopropyl)-24-[[(2S)-1-[[(2S)-1-[[(2S)-3-carboxy-1-[[(2S)-1-[[(2S,3R)-1-[[(2S)-1-[[(1S,2R)-1-carboxy-2-hydroxypropyl]amino]-1-oxo-3-phenylpropan-2-yl]amino]-3-hydroxy-1-oxobutan-2-yl]amino]-1-oxo-3-phenylpropan-2-yl]amino]-1-oxopropan-2-yl]amino]-3-(1H-indol-3-yl)-1-oxopropan-2-yl]amino]-1-oxopropan-2-yl]carbamoyl]-4,13-bis(2-carboxyethyl)-10,16,83-tris(carboxymethyl)-27,71-bis[(4-hydroxyphenyl)methyl]-54-(1H-imidazol-5-ylmethyl)-51-(2-methylpropyl)-33,74-bis(2-methylsulfanylethyl)-2,5,8,11,14,17,26,29,32,35,38,41,44,47,50,53,56,59,66,69,72,75,78,81,84,91-hexacosaoxo-21,22,62,63,87,88-hexathia-3,6,9,12,15,18,25,28,31,34,37,40,43,46,49,52,55,58,67,70,73,76,79,82,85,90-hexacosazatricyclo[43.40.4.219,60]hennonacontan-65-yl]amino]butanoic acid1525391: Inhibition of human Nav1.4 expressed in HEK293 cells by electrophysiology assayic500.1400uM
(4S)-4-[[(2S)-6-amino-2-[[(2S)-5-carbamimidamido-2-[[(1R,4S,7S,13S,16R,21R,24S,27S,30S,33S,36S,39S,42R,45S,48S,51S,54S,57S,60R,65R,68S,71S,74S)-27,51,74-tris(4-aminobutyl)-24-(3-amino-3-oxopropyl)-21-[[(2S)-2-[[(2S)-2-aminopent-4-ynoyl]amino]-3-(4-hydroxyphenyl)propanoyl]amino]-33-butyl-4,54-bis(3-carbamimidamidopropyl)-13-(2-carboxyethyl)-45-(carboxymethyl)-39-[(1R)-1-hydroxyethyl]-48-(hydroxymethyl)-30,36,68-tris(1H-indol-3-ylmethyl)-57-methyl-7,71-bis(2-methylpropyl)-3,6,9,12,15,22,25,28,31,34,37,40,43,46,49,52,55,58,67,70,73,76,81-tricosaoxo-18,19,62,63,78,79-hexathia-2,5,8,11,14,23,26,29,32,35,38,41,44,47,50,53,56,59,66,69,72,75,82-tricosazatricyclo[40.34.4.216,60]dooctacontane-65-carbonyl]amino]pentanoyl]amino]hexanoyl]amino]-5-[[(2S,3S)-1-amino-3-methyl-1-oxopentan-2-yl]amino]-5-oxopentanoic acid1363504: Inhibition of human Nav1.4 expressed in HEK293 cells at holding potential of -125 mV after 10 mins by patchxpress-based electrophysiology assayic500.1450uM
3-[4-(4-fluorophenoxy)phenyl]-N-methylpyrazole-1-carboxamide205290: Affinity for inactive human SkM1 sodium channel expressed in HEK293 cellski0.1500uM
(3S)-3-amino-4-[[(1R,4S,10S,13S,16S,19S,22S,25R,30R,33S,36S,39S,42S,45S,48S,51R,54S,57S,60S,63S,69S,72R,77R,80S,83S,86S,89S,92S,95S)-30-[[(2S)-6-amino-1-[[(2S)-1-[[(2S)-1-[[(2S)-1-amino-1-oxo-3-phenylpropan-2-yl]amino]-3-methyl-1-oxobutan-2-yl]amino]-3-(4-hydroxyphenyl)-1-oxopropan-2-yl]amino]-1-oxohexan-2-yl]carbamoyl]-22,36,95-tris(4-aminobutyl)-16,60-bis(2-amino-2-oxoethyl)-86-benzyl-4-[(2S)-butan-2-yl]-45,69,83,92-tetrakis(3-carbamimidamidopropyl)-13,19-bis(carboxymethyl)-42-[(1R)-1-hydroxyethyl]-48-(hydroxymethyl)-39-(1H-imidazol-5-ylmethyl)-33-(1H-indol-3-ylmethyl)-57,80-bis(2-methylpropyl)-89-(2-methylsulfanylethyl)-2,3a,5,11,14,17,20,23,32,35,38,41,44,47,50,53,56,59,62,68,71,78,81,84,87,90,93,96-octacosaoxo-54-propan-2-yl-a,27,28,74,75,99-hexathia-2a,3,6,12,15,18,21,24,31,34,37,40,43,46,49,52,55,58,61,67,70,79,82,85,88,91,94,97-octacosazapentacyclo[49.46.4.225,72.06,10.063,67]trihectan-77-yl]amino]-4-oxobutanoic acid1296072: Inhibition of human Nav1.4 expressed in HEK293 cells by whole cell patch clamp electrophysiology methodic500.1600uM
(4S)-4-[[(2S)-6-amino-2-[[(2S)-5-carbamimidamido-2-[[(1R,4S,7S,13S,16R,21R,24S,27S,30S,33S,36S,39S,42R,45S,48S,51S,54S,57S,60R,65R,68S,71S,74S)-27,51,74-tris(4-aminobutyl)-24-(3-amino-3-oxopropyl)-21-[[(2S)-2-[[(2S)-2-aminopent-4-ynoyl]amino]-3-(4-hydroxyphenyl)propanoyl]amino]-33-butyl-4,54-bis(3-carbamimidamidopropyl)-13-(2-carboxyethyl)-45-(carboxymethyl)-39-[(1R)-1-hydroxyethyl]-48-(hydroxymethyl)-30,36,68-tris(1H-indol-3-ylmethyl)-57-methyl-7,71-bis(2-methylpropyl)-3,6,9,12,15,22,25,28,31,34,37,40,43,46,49,52,55,58,67,70,73,76,81-tricosaoxo-18,19,62,63,78,79-hexathia-2,5,8,11,14,23,26,29,32,35,38,41,44,47,50,53,56,59,66,69,72,75,82-tricosazatricyclo[40.34.4.216,60]dooctacontane-65-carbonyl]amino]pentanoyl]amino]hexanoyl]amino]-5-[[(2S)-1-amino-1-oxo-3-phenylpropan-2-yl]amino]-5-oxopentanoic acid1363504: Inhibition of human Nav1.4 expressed in HEK293 cells at holding potential of -125 mV after 10 mins by patchxpress-based electrophysiology assayic500.1620uM
(3S)-3-amino-4-[[(1R,4S,10S,13S,16S,19S,22S,25R,30R,33S,36S,39S,42S,45S,48S,51R,54S,57S,60S,63S,69S,72R,77R,80S,86S,89S,92S,95S)-30-[[(2S)-6-amino-1-[[(2S)-1-[[(2S)-1-[[(2S)-1-amino-3-naphthalen-1-yl-1-oxopropan-2-yl]amino]-3-methyl-1-oxobutan-2-yl]amino]-3-(4-hydroxyphenyl)-1-oxopropan-2-yl]amino]-1-oxohexan-2-yl]carbamoyl]-22,36,95-tris(4-aminobutyl)-16,60-bis(2-amino-2-oxoethyl)-86-benzyl-4-[(2S)-butan-2-yl]-45,69,92-tris(3-carbamimidamidopropyl)-13,19-bis(carboxymethyl)-42-[(1R)-1-hydroxyethyl]-48-(hydroxymethyl)-39-(1H-imidazol-5-ylmethyl)-33-(1H-indol-3-ylmethyl)-57,80-bis(2-methylpropyl)-89-(2-methylsulfanylethyl)-2,3a,5,11,14,17,20,23,32,35,38,41,44,47,50,53,56,59,62,68,71,78,81,84,87,90,93,96-octacosaoxo-54-propan-2-yl-a,27,28,74,75,99-hexathia-2a,3,6,12,15,18,21,24,31,34,37,40,43,46,49,52,55,58,61,67,70,79,82,85,88,91,94,97-octacosazapentacyclo[49.46.4.225,72.06,10.063,67]trihectan-77-yl]amino]-4-oxobutanoic acid1296072: Inhibition of human Nav1.4 expressed in HEK293 cells by whole cell patch clamp electrophysiology methodic500.1700uM
(4S)-4-[[(2S)-6-amino-2-[[(2S)-5-carbamimidamido-2-[[(1R,4S,7S,13S,16R,21R,24S,27S,30S,33S,36S,39S,42R,45S,48S,51S,54S,57S,60R,65R,68S,71S,74S)-27,51,74-tris(4-aminobutyl)-24-(3-amino-3-oxopropyl)-21-[[(2S)-2-[[(2S)-2-aminopent-4-ynoyl]amino]-3-(4-hydroxyphenyl)propanoyl]amino]-33-butyl-4,54-bis(3-carbamimidamidopropyl)-13-(2-carboxyethyl)-45-(carboxymethyl)-71-cyclohexyl-39-[(1R)-1-hydroxyethyl]-48-(hydroxymethyl)-30,36,68-tris(1H-indol-3-ylmethyl)-57-methyl-7-(2-methylpropyl)-3,6,9,12,15,22,25,28,31,34,37,40,43,46,49,52,55,58,67,70,73,76,81-tricosaoxo-18,19,62,63,78,79-hexathia-2,5,8,11,14,23,26,29,32,35,38,41,44,47,50,53,56,59,66,69,72,75,82-tricosazatricyclo[40.34.4.216,60]dooctacontane-65-carbonyl]amino]pentanoyl]amino]hexanoyl]amino]-5-[[(2S,3S)-1-amino-3-methyl-1-oxopentan-2-yl]amino]-5-oxopentanoic acid1363504: Inhibition of human Nav1.4 expressed in HEK293 cells at holding potential of -125 mV after 10 mins by patchxpress-based electrophysiology assayic500.1770uM

CTD chemical–gene interactions

27 total (human), top 27 by PubMed support.

ChemicalActions (top 5)PubMed papers
bisphenol Aincreases expression, affects cotreatment, increases methylation2
chloroxylenoldecreases activity, affects activity, affects binding2
4-chlorophenolaffects activity, affects binding, decreases activity2
fluorene-9-bisphenolincreases expression1
pirinixic acidincreases activity, affects binding, decreases expression1
ethyl-p-hydroxybenzoatedecreases expression1
cypermethrindecreases expression1
tri-o-cresyl phosphateincreases expression1
2,6-xylenolaffects binding, decreases activity1
S-(1,2-dichlorovinyl)cysteinedecreases expression, decreases reaction1
3-cresolaffects activity1
CGP 52608increases reaction, affects binding1
2-methyl-4-chlorophenolaffects activity1
incobotulinumtoxinAdecreases expression1
N-(2-methyl-3-(4-(4-(4-(trifluoromethoxy)benzyloxy)piperidin-1-yl)-1,3,5-triazin-2-ylamino)phenyl)acetamidedecreases activity1
(+)-JQ1 compounddecreases expression1
Fulvestrantaffects cotreatment, increases methylation1
Troglitazoneincreases expression1
Benzo(a)pyrenedecreases methylation1
Cannabidioldecreases activity1
Diethylhexyl Phthalatedecreases expression1
Lidocaineaffects binding, decreases activity1
Lipopolysaccharidesdecreases expression, decreases reaction1
Valproic Acidincreases methylation1
1-Methyl-4-phenylpyridiniumincreases expression1
Okadaic Acidincreases expression1
Particulate Matterincreases methylation1

ChEMBL screening assays

95 unique, capped per target: 69 binding, 18 functional, 7 admet, 1 toxicity

Representative assays (with source publication via chembl_document):

Assay IDTypeDescriptionSource paper
CHEMBL1004181BindingInhibition of Nav1.4 ion channel assessed as reduction in cumulative sodium current2D- and 3D-QSAR of tocainide and mexiletine analogues acting as Na(v)1.4 channel blockers. — Eur J Med Chem
CHEMBL4040149ADMETInhibition of human NaV1.4 expressed in HEK cells assessed as half inactivation potential at -120 mV holding potential by automated patch clamp methodDiscovery of Clinical Candidate 4-[2-(5-Amino-1H-pyrazol-4-yl)-4-chlorophenoxy]-5-chloro-2-fluoro-N-1,3-thiazol-4-ylbenzenesulfonamide (PF-05089771): Design and Optimization of Diaryl Ether Aryl Sulfonamides as Selective Inhibitors of NaV1.7. — J Med Chem
CHEMBL6115363ToxicityInhibition of Nav1.4 (unknown origin) at 30 uM relative to controlDevelopment and characterization of pyridyl carboxamides as potent and highly selective Nav1.8 inhibitors. — Bioorg Med Chem Lett

Cellosaurus cell lines

2 cell lines: 1 spontaneously immortalized cell line, 1 transformed cell line

First 10 cell lines (id-ordered, not curated):

CellosaurusNameCategorySex
CVCL_C0YAB’SYS CHO Nav1.4Spontaneously immortalized cell lineFemale
CVCL_D1KJPrecisION hNav1.4-HEKTransformed cell lineFemale

Clinical trials (associated diseases)

311 trials via MONDO — disease-level, not drug-specific.

TrialPhaseStatusTitle
NCT02513940PHASE4COMPLETEDInfluence of Testosterone Administration on Drug-Induced QT Interval Prolongation and Torsades de Pointes
NCT03834883PHASE4COMPLETEDReducing the Risk of Drug-Induced QT Interval Lengthening in Women
NCT04169100PHASE4UNKNOWNNovel Form of Acquired Long QT Syndrome
NCT04675788PHASE4COMPLETEDNovel Approaches for Minimizing Drug-Induced QT Interval Lengthening
NCT00120055PHASE4COMPLETEDAssociation Between Systemic Exposure of Atorvastatin and Metabolites and Atorvastatin-induced Myotoxicity
NCT03633565PHASE4UNKNOWNComparative Study of Strategies for Management of Duchenne Myopathy (DM)
NCT00331656PHASE4UNKNOWNComparative Study of Non-Invasive Mask Ventilation vs Cuirass Ventilation in Patients With Acute Respiratory Failure.
NCT00994552PHASE4UNKNOWNComparison of Pressure Support and Pressure Control Ventilation in Chronic Respiratory Failure
NCT00004802PHASE3COMPLETEDPhase III Randomized, Double-Blind, Placebo-Controlled Study of Dichlorphenamide for Periodic Paralyses and Associated Sodium Channel Disorders
NCT01939561PHASE3COMPLETEDLamotrigine as Treatment of Myotonia
NCT02336477PHASE3COMPLETEDMexiletine and Non Dystrophic Myotonias
NCT00494507PHASE3COMPLETEDHyper- and Hypokalemic Periodic Paralysis Study
NCT06479135PHASE3RECRUITINGStudy of Navtemadlin add-on to Ruxolitinib in JAK Inhibitor-Naïve Patients With Myelofibrosis Who Have a Suboptimal Response to Ruxolitinib
NCT07317700PHASE3RECRUITINGA Clinical Trial of Flonoltinib Maleate for Intermediate or High-Risk Myelofibrosis
NCT03783923PHASE3TERMINATEDA Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
NCT06246513PHASE3ACTIVE_NOT_RECRUITINGA Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4
NCT01225614PHASE3UNKNOWNEfficacy and Tolerance of Early Launching of Nocturnal Non Invasive
NCT00839033PHASE3TERMINATEDEvaluation of a Mechanical Device During Acute Respiratory Failure in Patients With Neuromuscular Disorders
NCT00942227PHASE3COMPLETEDThe Value of Traction in Treatment of Lumbar Radiculopathy
NCT00979108PHASE3COMPLETEDThe Value of Traction in the Treatment of Cervical Radiculopathy
NCT01826487PHASE3COMPLETEDPhase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT02090959PHASE3TERMINATEDAn Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
NCT02436096PHASE3COMPLETEDA Study to Evaluate eFFIcacy and Safety of Sublingual TNX-102 SL Tablet Taken at Bedtime in Patients With fibRoMyalgia
NCT02829814PHASE3TERMINATEDRepeat of: A Study to Evaluate Efficacy and Safety of Sublingual TNX-102 SL Tablet Taken at Bedtime in Patients With Fibromyalgia
NCT03179631PHASE3COMPLETEDLong-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
NCT05126758PHASE3ACTIVE_NOT_RECRUITINGA Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
NCT05156320PHASE3COMPLETEDEfficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam
NCT05337553PHASE3ACTIVE_NOT_RECRUITINGA Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
NCT05626855PHASE3ACTIVE_NOT_RECRUITINGLong-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab
NCT06672237PHASE3RECRUITINGA Phase 3 Study of NTLA-2001 in ATTRv-PN
NCT02582476PHASE2TERMINATEDBumetanide in Hypokalaemic Periodic Paralysis
NCT07443306PHASE2RECRUITINGA Clinical Trial of Flonoltinib Maleate Tablets in the Treatment of JAK Inhibitor Refractory/Relapsed/Intolerant Patients With Medium to High Risk Myelofibrosis
NCT01648205PHASE2COMPLETEDLong-term Efficacy Study of Sodium Channel Blocker in LQT3 Patients
NCT02412709PHASE2UNKNOWNLong QT Syndrome Screening in Newborns
NCT04581408PHASE2COMPLETEDMutation-specific Therapy for the Long QT Syndrome
NCT04054375PHASE2COMPLETEDWeekly Steroids in Muscular Dystrophy
NCT01074359PHASE2TERMINATEDSafety and Efficacy Study of A0001 in Patients With the A3243G Mitochondrial DNA Point Mutation
NCT01371149PHASE2COMPLETEDPatient -Ventilator Interaction in Chronic Respiratory Failure
NCT02022072PHASE2TERMINATEDEvaluation of Vital Capacity
NCT03127514PHASE2COMPLETEDAMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS)

About this page

Generated deterministically by Sugi Atlas from primary biomedical databases via BioBTree. Every record on this page traces to a primary source.

Generated
Atlas version
v1.1.2
BioBTree
v2.0.2

Sources 81

This page pulls from 81 datasets indexed by BioBTree:

alphafoldalphamissenseantibodybgeebgee_evidencebindingdbbiogrid_interactionbrendabrenda_kineticsccdscellosauruschembl_activitychembl_assaychembl_documentchembl_moleculechembl_targetciviccivic_evidenceclingen_dosageclingen_gene_validityclinical_trialsclinvarcollectrictd_gene_interactiondbsnpdepmapdiamond_similarityefoensemblentrezesm2_similarityexonfantom5_genefantom5_promotergenccgenerifgogoparentgtopdbgtopdb_interactiongwasgwas_studyhgnchpointactinterprointogenjasparmeshmimmirdbmondomsigdborphanetorthologparalogpatentpatent_compoundpdbpfampharmgkb_clinicalpharmgkb_genepharmgkb_guidelinepharmgkb_variantpubchempubchem_activitypubchem_assaypubmedreactomereactomeparentrefseqrhearnacentralscxa_expressionscxa_gene_experimentsignorspliceaistring_interactiontranscriptufeatureuniprot