Nervous system disorder

Summary

Nervous system disorder (MONDO:0005071) is a disease (an umbrella term covering 72 Mondo subtypes) with 3 cohort genes (26 GWAS associations across 72 studies) and 442 clinical trials. Top therapeutic interventions include ataluren, botulinum toxin type a, and brexpiprazole.

At a glance

• Umbrella term: 72 Mondo subtypes
• Cohort genes: 3
• GWAS associations: 26
• Clinical trials: 442

Clinical features

No curated clinical features (Orphanet) for this disease.

Identifiers

Disease identifiers

Field	Value
Canonical name	nervous system disorder
Mondo ID	MONDO:0005071
EFO	EFO:0000618
MeSH	D009422
DOID	DOID:863
ICD-10-CM	G00-G99
NCIT	C26835
SNOMED CT	118940003
UMLS	C0027765
MedGen	14336
Anatomy (UBERON)	UBERON:0001016
Is cancer (heuristic)	no

Also known as: disease of nervous system · disease or disorder of nervous system · disorder of nervous system · nervous system disease · nervous system disease or disorder · nervous system disorder · neurologic disease · neurologic disorder · neurological disease · neurological disorder

Data availability: 26 GWAS associations (72 studies) · 3 GenCC gene-disease records · 8 cell lines.

Disease family

An umbrella term covering 72 Mondo subtypes.

Classification path: disease › human disease › disease by body system or component › nervous system disorder

Related subtypes (18): disorder of orbital region, integumentary system disorder, musculoskeletal system disorder, urinary system disorder, syndromic disease, auditory system disorder, breast disorder, connective tissue disorder, digestive system disorder, cardiovascular disorder, reproductive system disorder, immune system disorder, respiratory system disorder, endocrine system disorder, hematologic disorder, mouth disorder, disorder of visual system, otorhinolaryngologic disease

Subtypes (72): congenital nervous system disorder, central nervous system disorder, autoimmune disorder of the nervous system, cranial nerve neuropathy, peripheral nervous system disorder, neuronitis, diplegia of upper limb, retinal disorder, developmental disability, restless legs syndrome, movement disorder, toxic encephalopathy, Barre-Lieou syndrome, Gerstmann syndrome, drug-induced akathisia, drug-induced dyskinesia, stiff-person syndrome, Worster-Drought syndrome, corneal-cerebellar syndrome, pachygyria-intellectual disability-epilepsy syndrome, porencephaly-cerebellar hypoplasia-internal malformations syndrome, symmetrical thalamic calcifications, neonatal brainstem dysfunction, primary orthostatic hypotension, rippling muscle disease with myasthenia gravis, periodic paralysis, qualitative or quantitative protein defects in neuromuscular diseases, specific learning disability, cerebellar hypoplasia-tapetoretinal degeneration syndrome, locked-in syndrome, dopa-responsive dystonia, idiopathic recurrent stupor, chronic lymphocytic inflammation with pontine perivascular enhancement responsive to steroids, spontaneous periodic hypothermia, Sydenham chorea, duplication of the pituitary gland, Balint syndrome, paraneoplastic neurologic syndrome, persistent idiopathic facial pain, serotonin syndrome, hypothalamic adipsic hypernatraemia syndrome, exercise-induced malignant hyperthermia, perineural cyst, neuromuscular disease, neuromyelitis optica, AL amyloidosis, AA amyloidosis, neuroleptic malignant syndrome, infectious disorder of the nervous system, central nervous system malformation, synaptopathy, nervous system neoplasm, sensory ganglionopathy, radiculitis, wet beriberi, perceptual disorders, prepubertal anorexia nervosa, neurocutaneous syndrome, neurovascular disorder, Wallerian degeneration, nervous system injury, neurosarcoidosis, neuroendocrine disorder, tubulinopathy, atactic disorder, hereditary neurological disease, meningitis-retention syndrome, KIF1A related neurological disorder, neurological pain disorder, neurodevelopmental disorder, post 5-alpha-reductase inhibitors treatment syndrome, post-selective serotonin reuptake inhibitor sexual dysfunction

Genetics & variants

GWAS landscape

26 GWAS associations across 72 studies. Top hits map to 10 distinct genes (as reported by GWAS).

Top associations by p-value

rsID	p-value	Gene	Risk allele	Odds ratio
rs429358	8e-166	APOE	T	0.23
rs9367197	1e-12	CDC5L - SUPT3H	T	0.12
chr12:57132863	1e-12		A	0.04
rs763082055	2e-12	NXPH1 - GAPDHP68	G	2.05
chr1:3152348	2e-12		T	0.05
rs536642077	3e-12	HHAT	C	2.83
chr6:12903725	7e-12		G	0.04
rs183356941	1e-11	PTGFR, MGC27382	G	2.13
chr14:19059986	2e-10		C	2.41
chr5:91565525	2e-09		T	2.54
chr6:96617255	5e-09		T	0.04
rs143552363	9e-09	CAMTA1-IT1, CAMTA1	?
chr19:9908009	2e-08		T	2.61
chr14:57485510	2e-08		A	2.72
chr7:150993088	2e-08		T	0.06
chr3:91277237	3e-08		C	0.56
chr3:195093144	4e-08		G	1.24
chr10:43586637	5e-08		G	2.73
rs2150127	7e-08	GPC6	?
rs146329438	9e-08	NOPCHAP1	?
rs9584835	5e-07	FARP1	?
rs9613396	8e-07	LINC01638	?
rs139339493	9e-07	GRIK2 - R3HDM2P2	?

Top studies (by case count)

Study	Lead author	Year	Cases	Controls	Title
GCST90038600	Donertas HM	2021	83,484	401,114	Common genetic associations between age-related diseases.
GCST90473320	UK Biobank Whole-Genome Sequencing Consortium	2025	61,514	396,926	Whole-genome sequencing of 490,640 UK Biobank participants.
GCST90476506	Verma A	2024	58,508	357,691	Diversity and scale: Genetic architecture of 2068 traits in the VA Million Veteran Program.
GCST90038638	Donertas HM	2021	32,651	451,947	Common genetic associations between age-related diseases.
GCST90476505	Verma A	2024	15,108	97,035	Diversity and scale: Genetic architecture of 2068 traits in the VA Million Veteran Program.
GCST90473365	UK Biobank Whole-Genome Sequencing Consortium	2025	15,021	443,419	Whole-genome sequencing of 490,640 UK Biobank participants.
GCST90479139	Verma A	2024	7,586	427,112	Diversity and scale: Genetic architecture of 2068 traits in the VA Million Veteran Program.
GCST90038674	Donertas HM	2021	6,863	477,735	Common genetic associations between age-related diseases.
GCST90477589	Verma A	2024	6,739	437,670	Diversity and scale: Genetic architecture of 2068 traits in the VA Million Veteran Program.
GCST90435845	Zhou W	2018	4,655	402,383	Efficiently controlling for case-control imbalance and sample relatedness in large-scale genetic association studies.

Variant details and genetic-evidence tiers

Tier distribution (top 50 variants)

Tier	Variants
Tier 1: coding	1
Tier 2: splice/UTR	1
Tier 3: regulatory	0
Tier 4: intronic/intergenic	21

MAF distribution

Bucket	Variants
common (>=0.05)	5
low_freq (0.01-0.05)	0
rare (<0.01)	3
unknown	15

Functional consequences

Consequence	Count
unknown	12
intron_variant	6
intergenic_variant	3
missense_variant	1
3_prime_UTR_variant	1

Top variants

rsID	Chr	Pos	Alleles	MAF	Consequence	Gene	p-value	Tier
rs429358	19	44908684	T>C	0.139	missense_variant	APOE	8e-166	Tier 1: coding
rs9367197	6	44477709	T>C	0.412	intergenic_variant	CDC5L - SUPT3H	1e-12	Tier 4: intronic/intergenic
chr12:57132863							1e-12	Tier 4: intronic/intergenic
rs763082055	7	8790576	G>A,T	0.001	intergenic_variant	NXPH1 - GAPDHP68	2e-12	Tier 4: intronic/intergenic
chr1:3152348							2e-12	Tier 4: intronic/intergenic
rs536642077	1	210585455	C>T	0	intron_variant	HHAT	3e-12	Tier 4: intronic/intergenic
chr6:12903725							7e-12	Tier 4: intronic/intergenic
rs183356941	1	78364352	G>A	0.002	intron_variant	PTGFR, MGC27382	1e-11	Tier 4: intronic/intergenic
chr14:19059986							2e-10	Tier 4: intronic/intergenic
chr5:91565525							2e-09	Tier 4: intronic/intergenic
chr6:96617255							5e-09	Tier 4: intronic/intergenic
rs143552363	1	7354522	A>G		intron_variant	CAMTA1-IT1, CAMTA1	9e-09	Tier 4: intronic/intergenic
chr19:9908009							2e-08	Tier 4: intronic/intergenic
chr14:57485510							2e-08	Tier 4: intronic/intergenic
chr7:150993088							2e-08	Tier 4: intronic/intergenic
chr3:91277237							3e-08	Tier 4: intronic/intergenic
chr3:195093144							4e-08	Tier 4: intronic/intergenic
chr10:43586637							5e-08	Tier 4: intronic/intergenic
rs2150127	13	93838807	A>C,G,T	0.05	intron_variant	GPC6	7e-08	Tier 4: intronic/intergenic
rs146329438	12	105006104	A>G		3_prime_UTR_variant	NOPCHAP1	9e-08	Tier 2: splice/UTR
rs9584835	13	98402273	C>T	0.05	intron_variant	FARP1	5e-07	Tier 4: intronic/intergenic
rs9613396	22	27204866	C>T	0.05	intron_variant	LINC01638	8e-07	Tier 4: intronic/intergenic
rs139339493	6	102999105	T>G		intergenic_variant	GRIK2 - R3HDM2P2	9e-07	Tier 4: intronic/intergenic

Genes & proteins

Mendelian disease overlap and somatic drivers

GenCC: 6 · Orphanet: 0 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0

GenCC gene–disease validity (cohort genes)

the Disease column is the GenCC-asserted condition — a cohort gene’s strongest validity may be for a related predisposition syndrome.

Gene	Classification	Inheritance	Disease	Records
SKOR2	Limited	Autosomal recessive	nervous system disorder
TMEM92	Limited	Autosomal recessive	nervous system disorder
TOMM70	Limited	Autosomal dominant	nervous system disorder	4

Cohort genes → proteins

3 cohort genes, 3 distinct canonical proteins.

Evidence partition

Subset	Genes
multi_evidence	3

Cohort genes (full)

Symbol	HGNC	Ensembl	UniProt	Name	Evidence
TOMM70	HGNC:11985	ENSG00000154174	O94826	Mitochondrial import receptor subunit TOM70	gencc
TMEM92	HGNC:26579	ENSG00000167105	Q6UXU6	Transmembrane protein 92	gencc
SKOR2	HGNC:32695	ENSG00000215474	Q2VWA4	SKI family transcriptional corepressor 2	gencc

Cohort function summary

Lead sentence per gene, UniProt-curated.

Symbol	Protein name	Function (lead sentence)
TOMM70	Mitochondrial import receptor subunit TOM70	Acts as a receptor of the preprotein translocase complex of the outer mitochondrial membrane (TOM complex).
SKOR2	SKI family transcriptional corepressor 2	Exhibits transcriptional repressor activity.

Protein-family classification

Druggable: 0 · Difficult: 0 · Unknown: 3 · Druggable fraction: 0.0

Family distribution

Cohort families vs a genome-wide background (hypergeometric, BH-FDR; fold = observed/expected). Counts kept; sorted by enrichment, so the catch-all Other/Unknown bucket no longer leads.

Family	Genes	Fold	FDR
Other/Unknown	3	1.8×	0.174

Per-gene assignment

Symbol	Family	Druggable?	EC	InterPro (top 3)
TOMM70	Other/Unknown	no		TPR-like_helical_dom_sf, TPR_rpt
TMEM92	Other/Unknown	no		WBP1-like
SKOR2	Other/Unknown	no		SKI/SNO/DAC, DNA-bd_dom_put_sf, SAND-like_dom_sf

Expression context

Cohort genes with no expression data: 0.

3 cohort genes are a single-cell marker in ≥1 SCXA experiment.

Breadth distribution (Bgee present_calls)

Bucket	Genes
narrow (1-5 tissues)	0
moderate (6-20)	1
broad (>20)	2
unknown	0

Top tissues across cohort

Tissue	Cohort genes
Brodmann (1909) area 23	1
endothelial cell	1
gluteal muscle	1
duodenum	1
ileal mucosa	1
pancreatic ductal cell	1
hindlimb stylopod muscle	1
male germ line stem cell (sensu Vertebrata) in testis	1
sural nerve	1

Per-gene tissue summary (top 30)

Symbol	Bgee breadth	FANTOM5 breadth	SCXA	Top tissues
TOMM70	299	ubiquitous	marker	endothelial cell, gluteal muscle, Brodmann (1909) area 23
TMEM92	133	broad	marker	pancreatic ductal cell, ileal mucosa, duodenum
SKOR2	18		marker	male germ line stem cell (sensu Vertebrata) in testis, sural nerve, hindlimb stylopod muscle

Protein interactions among cohort

Intra-cohort edges: 0.

Hub genes (top 10 by interactor count)

Symbol	Interactor count
TOMM70	2,629
SKOR2	642
TMEM92	438

Structural data

PDB: 1 · AlphaFold-only: 2 · No structure: 0

Cohort genes with PDB structures (top 30)

Symbol	UniProt	PDB entries
TOMM70	O94826	3

AlphaFold-only cohort genes (top 30 by pLDDT)

Symbol	UniProt	pLDDT
TMEM92	Q6UXU6	67.18
SKOR2	Q2VWA4	52.95

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 6. Enrichment computed across 3 evidence-associated genes (1 with Reactome annotation).

Pathways by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 1 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

Pathway	Cohort genes	Fold	FDR	Sample cohort genes
PINK1-PRKN Mediated Mitophagy	1	356.9×	0.008	TOMM70
SARS-CoV-1 activates/modulates innate immune responses	1	271.9×	0.008	TOMM70
DDX58/IFIH1-mediated induction of interferon-alpha/beta	1	253.8×	0.008	TOMM70
Mitochondrial protein import	1	167.9×	0.009	TOMM70
SARS-CoV-2 activates/modulates innate and adaptive immune responses	1	89.2×	0.013	TOMM70
Ub-specific processing proteases	1	53.1×	0.019	TOMM70

GO biological processes by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 2 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

GO term	Cohort genes	Fold	FDR	Sample cohort genes
response to thyroxine	1	2808.7×	0.005	TOMM70
regulation of cerebellar granule cell precursor proliferation	1	2106.5×	0.005	SKOR2
regulation of neuroblast proliferation	1	1685.2×	0.005	SKOR2
negative regulation of cell growth involved in cardiac muscle cell development	1	702.2×	0.006	TOMM70
protein insertion into mitochondrial inner membrane	1	648.1×	0.006	TOMM70
protein insertion into mitochondrial outer membrane	1	648.1×	0.006	TOMM70
cerebellar Purkinje cell differentiation	1	526.6×	0.006	SKOR2
cell development	1	443.5×	0.006	SKOR2
regulation of dendrite morphogenesis	1	366.4×	0.007	SKOR2
protein import into mitochondrial matrix	1	351.1×	0.007	TOMM70
obsolete protein targeting to mitochondrion	1	290.6×	0.007	TOMM70
positive regulation of defense response to virus by host	1	263.3×	0.007	TOMM70
obsolete positive regulation of protein targeting to mitochondrion	1	247.8×	0.007	TOMM70
activation of innate immune response	1	240.7×	0.007	TOMM70
positive regulation of smoothened signaling pathway	1	210.7×	0.007	SKOR2
positive regulation of interferon-beta production	1	195.9×	0.007	TOMM70
negative regulation of BMP signaling pathway	1	145.3×	0.009	SKOR2
cellular response to virus	1	100.3×	0.013	TOMM70
smoothened signaling pathway	1	90.6×	0.013	SKOR2
negative regulation of transforming growth factor beta receptor signaling pathway	1	86.9×	0.013	SKOR2
regulation of apoptotic process	1	41.7×	0.026	TOMM70
regulation of DNA-templated transcription	1	15.8×	0.065	SKOR2
negative regulation of transcription by RNA polymerase II	1	8.9×	0.110	SKOR2

Therapeutics

Drugs indicated or in trials for this disease

No drug has an approved disease-direct ChEMBL indication for this disease.

6 drugs in clinical trials for this disease (phase 2–3, investigational): efficacy not established — a trial record, not an indication.

Drug	Highest phase
Onabotulinumtoxina	Phase 3
Alteplase	Phase 2
Dexamethasone	Phase 2
Ferumoxytol	Phase 2
Tenecteplase	Phase 2
Tianeptine	Phase 2

Drug target analysis

Approved (phase 4): 0 · Phase ≥3: 0 · Phased (≥1): 0 · Undrugged: 3

Druggability breadth: 1 of 3 evidence-associated genes (33%) have a ChEMBL target (buckets above are over the deeply-mined display cohort).

Top cohort targets by molecule count

Symbol	Molecules	Max phase
TOMM70	0	0
TMEM92	0	0
SKOR2	0	0

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 0.

Cohort genes with ChEMBL bioactivity (full, sorted by assay count)

Symbol	Assays	Type breakdown
TOMM70	2	Binding:2

Pharmacogenomics

Cohort genes with a PharmGKB record: 3; with CPIC/DPWG dosing guidelines: 0.

No cohort gene has a CPIC/DPWG genotype-guided dosing guideline (PharmGKB).

Chemical tractability of cohort targets

0 approved/phased compounds have measured bioactivity against a cohort gene (and aren’t yet in disease-level trials). This is a research / tractability signal, NOT a therapeutic recommendation — a bioactivity row often reflects off-target or screening binding (e.g. promiscuous kinase inhibitors against a cohort kinase), implying no disease mechanism.

Druggability pyramid

Cohort genes binned by druggability tier (high → low):

Tier	Definition	Genes	Symbols
A	Approved (phase 4 drug)	0
B	Phased (≥1) drug, not yet approved	0
C	Druggable family + PDB, no drug	0
D	Druggable family + AlphaFold only, no drug	0
E	Difficult family or no structure, no drug	3	TOMM70, TMEM92, SKOR2

Undrugged target profiles

3 cohort genes are undrugged. Ranked by ‘starting-point quality’ (assay depth + drugged-partner adjacency).

Symbol	ChEMBL assays	Drugged partners (top 3)
TOMM70	2	—
TMEM92	0	—
SKOR2	0	—

Clinical trials & evidence

Clinical trials

Clinical trials: 442.

Phase distribution (across all retrieved trials)

Phase	Trials
Not specified	354
PHASE3	26
PHASE2	25
PHASE1	12
PHASE4	11
PHASE1/PHASE2	10
EARLY_PHASE1	3
PHASE2/PHASE3	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT04289142	PHASE4	RECRUITING	Cognitive Outcomes After Dexmedetomidine Sedation in Cardiac Surgery Patients
NCT06406127	PHASE4	RECRUITING	Effect of Alpha Lipoic Acid on Chemotherapy Induced Neurological Changes in Breast Cancer Patients
NCT00560157	PHASE4	COMPLETED	Nutritional and Metabolic Evaluation of a Tube Feeding Immune Enhancing Diet in ICU Patients
NCT01319643	PHASE4	UNKNOWN	Normal Oxygenation Versus Hyperoxia in the Intensive Care Unit (ICU)
NCT01662414	PHASE4	COMPLETED	Effect of Undenatured Cysteine-Rich Whey Protein Isolate (HMS 90®) in Patients With Parkinson’s Disease
NCT04386525	PHASE4	UNKNOWN	Omega 3 and Ischemic Stroke; Fish Oil as an Option
NCT04871464	PHASE4	UNKNOWN	Role and Mechanism of Probiotics in Improving Motor Symptoms in Mild to Moderate Parkinson’s Disease
NCT04970667	PHASE4	COMPLETED	Flupentixol and Melitracen Tablets in the Treatment of Emotional Disorder
NCT05068349	PHASE4	UNKNOWN	For Patients With Ischemic Stroke, Clinically Study the Effectiveness and Safety of Butylphthalide.
NCT05657860	PHASE4	COMPLETED	Guanfacine Extended Release for the Reduction of Aggression and Self-injurious Behavior Associated With Prader-Willi Syndrome
NCT06382467	PHASE4	UNKNOWN	Comparison of Remimazolam and Propofol Combination vs. Propofol in IOM
NCT05126758	PHASE3	ACTIVE_NOT_RECRUITING	A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
NCT05508789	PHASE3	ACTIVE_NOT_RECRUITING	A Study of Donanemab (LY3002813) in Participants With Early Symptomatic Alzheimer’s Disease (TRAILBLAZER-ALZ 5)
NCT05738486	PHASE3	ACTIVE_NOT_RECRUITING	A Study of Different Donanemab (LY3002813) Dosing Regimens in Adults With Early Alzheimer’s Disease (TRAILBLAZER-ALZ 6)
NCT05834855	PHASE3	RECRUITING	Non-inferiority Study of Rituximab Compared to Ocrelizumab in Relapsing MS
NCT06672237	PHASE3	RECRUITING	A Phase 3 Study of NTLA-2001 in ATTRv-PN
NCT06819592	PHASE3	RECRUITING	PRophylaxis Against Early VENTilator-associated Infections in Acute Brain Injury
NCT07587242	PHASE3	NOT_YET_RECRUITING	A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping
NCT00240695	PHASE3	COMPLETED	A Follow-up Study to Assess Safety and Tolerability of Galantamine Treatment in Individuals With Mild Cognitive Impairment
NCT00532571	PHASE2/PHASE3	COMPLETED	Effects of Coenzyme Q10 in PSP and CBD
NCT01313299	PHASE3	COMPLETED	Dysport® Adult Upper Limb Spasticity
NCT01313312	PHASE3	COMPLETED	Dysport® Adult Upper Limb Spasticity Extension Study
NCT01425983	PHASE3	COMPLETED	Dietary Intervention of Stress-Induced Neurovegetative Disorders With a Specific Amino Acid Composition (asn01)
NCT01589289	PHASE3	COMPLETED	Rapid Diagnostic Tests and Clinical/Laboratory Predictors of Tropical Diseases in Neurological Disorders in DRC
NCT01826487	PHASE3	COMPLETED	Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT01862640	PHASE3	COMPLETED	A Phase 3, 12-week, Multicenter, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Efficacy, Safety, and Tolerability of 2 Fixed Doses of Brexpiprazole in the Treatment of Alzheimer’s Agitation
NCT01922258	PHASE3	COMPLETED	Safety and Tolerability Study of Flexible Dosing of Brexpiprazole in the Treatment of Subjects With Agitation Associated With Dementia of the Alzheimer’s Type
NCT02090959	PHASE3	TERMINATED	An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
NCT02284126	PHASE3	COMPLETED	Topical Vancomycin for Neurosurgery Wound Prophylaxis
NCT02436096	PHASE3	COMPLETED	A Study to Evaluate eFFIcacy and Safety of Sublingual TNX-102 SL Tablet Taken at Bedtime in Patients With fibRoMyalgia
NCT02770807	PHASE3	COMPLETED	Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Telangiectasia Patients
NCT02829814	PHASE3	TERMINATED	Repeat of: A Study to Evaluate Efficacy and Safety of Sublingual TNX-102 SL Tablet Taken at Bedtime in Patients With Fibromyalgia
NCT03179631	PHASE3	COMPLETED	Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
NCT03336450	PHASE3	COMPLETED	Study of Midazolam Hydrochloride Oromucosal Solution (MHOS/SHP615) in Pediatric Patients With Status Epilepticus (Convulsive) in the Community Setting
NCT03336645	PHASE3	COMPLETED	Open-label Study of Midazolam Hydrochloride Oromucosal Solution (MHOS/SHP615) in Children With Status Epilepticus (Convulsive) in a Healthcare Setting in Japan
NCT04639310	PHASE3	TERMINATED	XEN496 (Ezogabine) in Children With KCNQ2 Developmental and Epileptic Encephalopathy
NCT04912856	PHASE3	TERMINATED	An Open-Label Extension of the Study XEN496 (Ezogabine) in Children With KCNQ2-DEE
NCT05361707	PHASE3	UNKNOWN	Evaluating the Effects of Tasimelteon in Individuals With Autism Spectrum Disorder (ASD) and Sleep Disturbances
NCT04460872	PHASE2	RECRUITING	Locomotor Training With Testosterone to Promote Bone and Muscle Health After Spinal Cord Injury
NCT04684602	PHASE1/PHASE2	RECRUITING	Mesenchymal Stem Cells for the Treatment of Various Chronic and Acute Conditions

Drugs tested across these trials (top 30)

Molecule	Max phase	Trials referencing
ATALUREN	4	3
BOTULINUM TOXIN TYPE A	4	2
BREXPIPRAZOLE	4	2
CEFTRIAXONE	4	2
DONANEMAB	4	2
EZOGABINE	4	2
LEVOMEFOLIC ACID	4	2
MIDAZOLAM HYDROCHLORIDE	4	2
OCRELIZUMAB	4	2
TESTOSTERONE ENANTHATE	4	2
ALTEPLASE	4	1
BETAINE	4	1
CENOBAMATE	4	1
CYANOCOBALAMIN	4	1
FERUMOXYTOL	4	1
FINASTERIDE	4	1
FLORBETABEN F18	4	1
GALANTAMINE HYDROBROMIDE	4	1
OLIVE OIL	4	1
OXYGEN	4	1
PERAMPANEL	4	1
REMIMAZOLAM BESYLATE	4	1
ROTIGOTINE	4	1
TENECTEPLASE	4	1
RAC-3-N-BUTYLPHTHALIDE	3	2
CREATINE	3	1
FLUPENTIXOL	3	1
LEUCINE	3	1
MELITRACEN	3	1
OMEGA-3 FATTY ACIDS	3	1