TUBB4A

Summary

TUBB4A (tubulin beta 4A class IVa, HGNC:20774) is a protein-coding gene on chromosome 19p13.3, encoding Tubulin beta-4A chain (P04350). Tubulin is the major constituent of microtubules, a cylinder consisting of laterally associated linear protofilaments composed of alpha- and beta-tubulin heterodimers.

This gene encodes a member of the beta tubulin family. Beta tubulins are one of two core protein families (alpha and beta tubulins) that heterodimerize and assemble to form microtubules. Mutations in this gene cause hypomyelinating leukodystrophy-6 and autosomal dominant torsion dystonia-4. Alternate splicing results in multiple transcript variants encoding different isoforms. A pseudogene of this gene is found on chromosome X.

Source: NCBI Gene 10382 — RefSeq curated summary.

At a glance

• Gene–disease (curated): TUBB4A-related neurologic disorder (Definitive, ClinGen) — +2 more curated relationships
• GWAS associations: 2
• Clinical variants (ClinVar): 355 total — 8 pathogenic, 24 likely-pathogenic
• Phenotypes (HPO): 56
• Druggable target: yes — 21 molecules with ChEMBL bioactivity
• Dosage sensitivity (ClinGen): haploinsufficiency no evidence, triplosensitivity no evidence
• MANE Select transcript: NM_006087

Identifiers

Gene identifiers

Field	Value
HGNC ID	HGNC:20774
Approved symbol	TUBB4A
Name	tubulin beta 4A class IVa
Location	19p13.3
Locus type	gene with protein product
Status	Approved
Aliases	beta-5
Ensembl gene	ENSG00000104833
Ensembl biotype	protein_coding
OMIM	602662
Entrez	10382

Gene structure

Transcript identifiers

Ensembl transcripts: 13 — 11 protein_coding, 2 nonsense_mediated_decay

ENST00000264071, ENST00000594075, ENST00000594276, ENST00000594290, ENST00000595324, ENST00000596291, ENST00000596926, ENST00000597686, ENST00000598006, ENST00000598635, ENST00000601152, ENST00000601640, ENST00000714086

RefSeq mRNA: 6 — MANE Select: NM_006087 NM_001289123, NM_001289127, NM_001289129, NM_001289130, NM_001289131, NM_006087

CCDS: CCDS12168

Canonical transcript exons

ENST00000264071 — 4 exons

Exon	Start	End
ENSE00001159938	6494319	6496221
ENSE00001630000	6501515	6501623
ENSE00003206964	6502156	6502309
ENSE00003464353	6501287	6501397

Expression profiles

Bgee: expression breadth ubiquitous, 201 present calls, max score 99.86.

FANTOM5 (CAGE): breadth broad, TPM avg 48.4537 / max 4721.8513, expressed in 770 samples.

FANTOM5 promoters (6 alternative TSS)

Promoter ID	TPM avg	Samples expressed
178685	44.3657	721
178686	3.6923	434
178684	0.1334	63
178687	0.1203	62
178688	0.0910	44
178689	0.0510	35

Top tissues by expression

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

Tissue	Anatomy ID	Expression score	Quality
right hemisphere of cerebellum	UBERON:0014890	99.86	gold quality
cerebellar hemisphere	UBERON:0002245	99.83	gold quality
cerebellar cortex	UBERON:0002129	99.82	gold quality
Brodmann (1909) area 10	UBERON:0013541	99.72	gold quality
cerebellum	UBERON:0002037	99.60	gold quality
C1 segment of cervical spinal cord	UBERON:0006469	99.59	gold quality
right frontal lobe	UBERON:0002810	99.57	gold quality
paraflocculus	UBERON:0005351	99.55	gold quality
prefrontal cortex	UBERON:0000451	99.51	gold quality
middle frontal gyrus	UBERON:0002702	99.38	gold quality
spinal cord	UBERON:0002240	99.22	gold quality
cortical plate	UBERON:0005343	99.20	gold quality
cingulate cortex	UBERON:0003027	99.10	gold quality
anterior cingulate cortex	UBERON:0009835	99.10	gold quality
amygdala	UBERON:0001876	98.97	gold quality
caudate nucleus	UBERON:0001873	98.93	gold quality
medial globus pallidus	UBERON:0002477	98.92	gold quality
hypothalamus	UBERON:0001898	98.88	gold quality
nucleus accumbens	UBERON:0001882	98.79	gold quality
inferior vagus X ganglion	UBERON:0005363	98.72	gold quality
frontal cortex	UBERON:0001870	98.67	gold quality
frontal lobe	UBERON:0016525	98.67	gold quality
dorsolateral prefrontal cortex	UBERON:0009834	98.61	gold quality
putamen	UBERON:0001874	98.39	gold quality
globus pallidus	UBERON:0001875	98.37	gold quality
postcentral gyrus	UBERON:0002581	98.29	gold quality
cerebellar vermis	UBERON:0004720	98.23	gold quality
corpus callosum	UBERON:0002336	98.21	gold quality
Brodmann (1909) area 9	UBERON:0013540	98.17	gold quality
left adrenal gland cortex	UBERON:0035825	98.17	gold quality

Single-cell (SCXA)

Detected in 8 experiment(s), a significant marker in 4.

Experiment	Marker?	Max mean expression
E-GEOD-36552	yes	1513.77
E-MTAB-6819	yes	557.73
E-GEOD-84465	yes	14.22
E-GEOD-93593	yes	6.98
E-MTAB-3929	no	2740.21
E-GEOD-81383	no	196.15
E-MTAB-6386	no	8.80
E-ANND-3	no	3.30

Regulation

Is transcription factor: no

miRNA regulators (miRDB)

66 targeting TUBB4A, top 30 by miRDB confidence (max_score; target_count = how many genes the miRNA targets in total — lower means more specific):

miRNA	Max score	Avg score	miRNA target_count
HSA-MIR-4283	100.00	66.42	2097
HSA-MIR-6833-3P	100.00	70.63	3197
HSA-MIR-6127	100.00	66.76	2188
HSA-MIR-6129	100.00	66.46	2080
HSA-MIR-520G-5P	99.99	66.76	658
HSA-MIR-4500	99.99	72.72	2367
HSA-MIR-6845-3P	99.94	66.88	1439
HSA-MIR-4731-5P	99.89	67.23	2537
HSA-MIR-4447	99.85	67.81	2900
HSA-MIR-4728-5P	99.85	69.39	4718
HSA-MIR-4739	99.84	65.25	1832
HSA-MIR-1321	99.84	65.30	1811
HSA-MIR-4756-5P	99.84	64.98	1809
HSA-MIR-6785-5P	99.82	68.68	4428
HSA-MIR-577	99.78	69.13	2479
HSA-MIR-149-3P	99.72	68.22	3963
HSA-MIR-10393-3P	99.72	66.56	961
HSA-MIR-6801-5P	99.72	66.50	981
HSA-MIR-6883-5P	99.69	68.05	3785
HSA-MIR-3934-5P	99.67	64.04	846
HSA-MIR-4527	99.66	67.43	714
HSA-MIR-1827	99.63	68.57	3265
HSA-MIR-6503-5P	99.62	66.96	597
HSA-MIR-4756-3P	99.62	66.30	1319
HSA-MIR-6797-5P	99.61	66.55	2084
HSA-MIR-1249-5P	99.61	66.55	2049
HSA-MIR-4472	99.56	66.08	1478
HSA-MIR-6716-5P	99.56	68.62	1244
HSA-MIR-4437	99.52	65.29	1266
HSA-MIR-1207-5P	99.49	69.11	2983

Functional genomics

ClinGen dosage: haploinsufficiency 0 (no evidence), triplosensitivity 0 (no evidence). ClinGen Gene Dosage Map

Literature-anchored findings (GeneRIF, showing 37)

• The results provide the first evidence that a specific isoform of beta-tubulin is required for mitosis. (PMID:18553364)
• Suggest that nuclear accumulation of soluble tubulin is part of an intrinsic defense mechanism, which tends to limit cell proliferation under pathological conditions. (PMID:19299461)
• Data show six differentially expressed proteins were identified as HSP70, PPIA and alpha-Enolase (up-regulated) S100-A9, PIMT and beta-5 tubulin (down-regulated), most of which had been shown to play a potential role in the pathogenesis of atherosclerosis. (PMID:21839816)
• DYT4 is a familial form of dystonia unrelated to known dystonia genes and loci. Phenotypic expression is variable, ranging from isolated spasmodic dysphonia (often with mild craniocervical dystonia) to severe generalized dystonia. (PMID:21956287)
• Destruction of the beta-tubulin:CCT-beta complex provokes Hsp90-dependent protein ubiquitination and degradation. (PMID:23190606)
• This study demonistrated that TUBB4A mutation in the autoregulatory domain cause hereditary dystonia. (PMID:23424103)
• A de novo mutation in the beta-tubulin gene TUBB4A results in the leukoencephalopathy hypomyelination with atrophy of the basal ganglia and cerebellum. (PMID:23582646)
• This study provided strong evidence supporting the causative role of a mutation in TUBB4, altering a highly conserved and functionally important amino acid, in DYT4 dystonia. (PMID:23595291)
• Data indicate that leucine-rich repeat kinase 2 (LRRK2) selectively interacts with three beta-tubulin isoforms: TUBB, TUBB4, and TUBB6. (PMID:24275654)
• The c.4C>G DYT4 mutation appears to be private, and clinical testing for TUBB4A mutations is not justified in spasmodic dysphonia or other forms of primary dystonia (PMID:24598712)
• Novel TUBB4A mutations and expansion of the neuroimaging phenotype of hypomyelination with atrophy of the basal ganglia and cerebellum (H-ABC). (PMID:24706558)
• This study demonistrated that Hypomyelination with atrophy of the basal ganglia and cerebellum with TUBB4A mutation. (PMID:24785942)
• TUBB4A mutations cause typical hypomyelinating leukoencephalopathies. (PMID:24850488)
• Novel TUBB4A mutations expands the phenotype of TUBB4A-related hypomyelinating conditions beyond hypomyelination with atrophy of the basal ganglia and cerebellum. (PMID:25085639)
• Data suggested H-ABC and DYT4 belong to a continuous phenotypic spectrum associated with TUBB4A mutations. (PMID:25545912)
• The study adds complicated hereditary spastic paraplegia to the clinical spectrum of TUBB4A-associated neurological disorders. (PMID:25772097)
• our data indicate that TUBB4A coding mutations do not play a critical role in the broad population of isolated dystonia patients (PMID:26318963)
• a paclitaxel-resistant beta-tubulin isotype, betaIVa-tubulin, was the most up-regulated gene compared with other beta-tubulin isotypes in H460 floating cells, concomitant with elevated ERK activation (PMID:26375501)
• TUBB4A-mutated patients manifest a comparable clinical and neuroimaging picture but they can differ from each other in terms of rate of disease progression (PMID:26643067)
• Genetic screening targeted at currently known disease-causing mutations in TOR1A, THAP1, and TUBB4 appears to have low diagnostic yield in sporadic spasmodic dysphonia. In our cohort, only 2 patients tested positive for novel/rare variants in THAP1. (PMID:27188707)
• TUBB3 and TUBB4 are necessary for the transport and proper localization of N-cadherin within the plasma membrane. (PMID:28648944)
• The data of this studies suggest that mutations in TUBB4A exceedingly rarely contribute to the etiology of isolated dystonia. (PMID:28655586)
• The different clinical phenotypes associated with TUBB4A reflect the selective and specific cellular effects of the causative mutations. Cellular specificity of disease pathogenesis is relevant to developing targeted treatments for this disabling condition. (PMID:28973395)
• Together, DYT4-associated TUBB4A mutants themselves form aberrant tubulin networks and inhibit neuronal process growth, possibly explaining progress through the pathological states at cellular levels. (PMID:29127012)
• In summary, betaV-tubulin was localized to secretory cells of the distal Fallopian tube epithelium and its expression varied according to the clinical diagnosis. (PMID:29298171)
• Neuroblastoma cells expressing TUBB4A mutations p.D249N and p.A271T showed reduced neurite outgrowth, less dynamic microtubules, and an altered Kif-5 binding affinity. Endogenously mutated induced pluripotent stem cell (iPSC)-derived neurons from patients and in CRISPR/Cas9-derived heterozygous TUBB4A knockout iPSC-derived neurons, we detected alterations in mitochondrial transport. (PMID:30079973)
• Estimating the relative frequency of leukodystrophies and recommendations for carrier screening in the era of next-generation sequencing. (PMID:32573057)
• Corpus callosum thinning in autosomal dominant hereditary spastic paraplegia associated with a novel TUBbeta4A mutation. (PMID:32720309)
• DYT-TUBB4A (DYT4 Dystonia): New Clinical and Genetic Observations. (PMID:32943487)
• Acquisition of Developmental Milestones in Hypomyelination With Atrophy of the Basal Ganglia and Cerebellum and Other TUBB4A-Related Leukoencephalopathy. (PMID:34514881)
• In-silico phenotype prediction by normal mode variant analysis in TUBB4A-related disease. (PMID:34997144)
• H-ABC- and dystonia-causing TUBB4A mutations show distinct pathogenic effects. (PMID:35275727)
• TUBB4A interacts with MYH9 to protect the nucleus during cell migration and promotes prostate cancer via GSK3beta/beta-catenin signalling. (PMID:35589707)
• Novel TUBB4A mutation in a family with hereditary spastic paraplegia. (PMID:35871212)
• [Analysis of TUBB4A gene variant in a patient with adolescent-onset hypomyelinating leukodystrophy with atrophy of basal ganglia and cerebellum]. (PMID:36972930)
• Mutation in the beta-tubulin gene TUBB4A results in epileptic encephalopathy associated with hypomyelinated leucodystrophy: Unexpected findings reveal genetic mosaicism. (PMID:37529938)
• Expanding the phenotypic and genotypic spectrum of DYT-TUBB4A with seven patients from India. (PMID:38762926)

Cross-species orthologs

2 orthologs

Organism	Symbol	Gene ID
mus_musculus	Tubb4a	ENSMUSG00000062591
rattus_norvegicus	Tubb4a	ENSRNOG00000047505

Paralogs (23): TUBG2 (ENSG00000037042), TUBE1 (ENSG00000074935), TUBA3D (ENSG00000075886), TUBB1 (ENSG00000101162), TUBD1 (ENSG00000108423), TUBA1B (ENSG00000123416), TUBA4A (ENSG00000127824), TUBG1 (ENSG00000131462), TUBB2A (ENSG00000137267), TUBB2B (ENSG00000137285), TUBA3E (ENSG00000152086), TUBA1A (ENSG00000167552), TUBA1C (ENSG00000167553), TUBB8B (ENSG00000173213), TUBB6 (ENSG00000176014), TUBAL3 (ENSG00000178462), TUBA8 (ENSG00000183785), TUBB4B (ENSG00000188229), TUBB (ENSG00000196230), TUBA3C (ENSG00000198033), TUBA4B (ENSG00000243910), TUBB3 (ENSG00000258947), TUBB8 (ENSG00000261456)

Protein

Protein identifiers

Tubulin beta-4A chain — P04350 (reviewed: P04350)

Alternative names: Tubulin 5 beta, Tubulin beta-4 chain

All UniProt accessions (13): A0AAQ5BHG7, P04350, M0QX14, M0QY37, M0QY85, M0QYM7, M0QZL7, M0R0M1, M0R0X0, M0R1I1, M0R278, M0R2D3, M0R2T4

UniProt curated annotations — full annotation on UniProt →

Function. Tubulin is the major constituent of microtubules, a cylinder consisting of laterally associated linear protofilaments composed of alpha- and beta-tubulin heterodimers. Microtubules grow by the addition of GTP-tubulin dimers to the microtubule end, where a stabilizing cap forms. Below the cap, tubulin dimers are in GDP-bound state, owing to GTPase activity of alpha-tubulin.

Subunit / interactions. Dimer of alpha and beta chains. A typical microtubule is a hollow water-filled tube with an outer diameter of 25 nm and an inner diameter of 15 nM. Alpha-beta heterodimers associate head-to-tail to form protofilaments running lengthwise along the microtubule wall with the beta-tubulin subunit facing the microtubule plus end conferring a structural polarity. Microtubules usually have 13 protofilaments but different protofilament numbers can be found in some organisms and specialized cells.

Subcellular location. Cytoplasm. Cytoskeleton.

Tissue specificity. Major isotype in brain, where it represents 46% of all beta-tubulins. In the brain, highest expression levels in the cerebellum, followed by putamen and white matter. Moderate levels in testis. Very low levels, if any, in other tissues.

Post-translational modifications. Some glutamate residues at the C-terminus are polyglutamylated, resulting in polyglutamate chains on the gamma-carboxyl group. Polyglutamylation plays a key role in microtubule severing by spastin (SPAST). SPAST preferentially recognizes and acts on microtubules decorated with short polyglutamate tails: severing activity by SPAST increases as the number of glutamates per tubulin rises from one to eight, but decreases beyond this glutamylation threshold. Glutamylation is also involved in cilia motility. Some glutamate residues at the C-terminus are monoglycylated but not polyglycylated due to the absence of functional TTLL10 in human. Monoglycylation is mainly limited to tubulin incorporated into cilia and flagella axonemes, which is required for their stability and maintenance. Flagella glycylation controls sperm motility. Both polyglutamylation and monoglycylation can coexist on the same protein on adjacent residues, and lowering glycylation levels increases polyglutamylation, and reciprocally. Phosphorylated on Ser-172 by CDK1 during the cell cycle, from metaphase to telophase, but not in interphase. This phosphorylation inhibits tubulin incorporation into microtubules.

Disease relevance. Dystonia 4, torsion, autosomal dominant (DYT4) [MIM:128101] A form of torsion dystonia, a disease defined by the presence of sustained involuntary muscle contractions, often leading to abnormal postures. ‘Torsion’ refers to the twisting nature of body movements, often affecting the trunk. DYT4 is characterized by onset in the second to third decade of progressive laryngeal dysphonia followed by the involvement of other muscles, such as the neck or limbs. Some patients develop an ataxic gait. The disease is caused by variants affecting the gene represented in this entry. Leukodystrophy, hypomyelinating, 6 (HLD6) [MIM:612438] A neurologic disorder characterized by onset in infancy or early childhood of delayed motor development and gait instability, followed by extrapyramidal movement disorders, such as dystonia, choreoathetosis, rigidity, opisthotonus, and oculogyric crises, progressive spastic tetraplegia, ataxia, and, more rarely, seizures. Most patients have cognitive decline and speech delay, but some can function normally. Brain MRI shows a combination of hypomyelination, cerebellar atrophy, and atrophy or disappearance of the putamen. The disease is caused by variants affecting the gene represented in this entry.

Domain organisation. The highly acidic C-terminal region may bind cations such as calcium. The MREI motif is common among all beta-tubulin isoforms and may be critical for tubulin autoregulation.

Similarity. Belongs to the tubulin family.

RefSeq proteins (6): NP_001276052, NP_001276056, NP_001276058, NP_001276059, NP_001276060, NP_006078* (*=MANE)

Domains & families (InterPro)

ID	Name	Type
IPR000217	Tubulin	Family
IPR002453	Beta_tubulin	Family
IPR003008	Tubulin_FtsZ_GTPase	Domain
IPR008280	Tub_FtsZ_C	Homologous_superfamily
IPR013838	Beta-tubulin_BS	Binding_site
IPR017975	Tubulin_CS	Conserved_site
IPR018316	Tubulin/FtsZ_2-layer-sand-dom	Domain
IPR023123	Tubulin_C	Homologous_superfamily
IPR036525	Tubulin/FtsZ_GTPase_sf	Homologous_superfamily
IPR037103	Tubulin/FtsZ-like_C	Homologous_superfamily

Pfam: PF00091, PF03953

UniProt features (20 total): binding site 9, sequence variant 4, sequence conflict 3, modified residue 2, chain 1, short sequence motif 1

Structure

Experimental structures (PDB)

0 structures.

Predicted structure (AlphaFold)

Model	pLDDT	Fraction very-high
AF-P04350-F1	92.25	0.84

Functional residue map

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

Ligand- & substrate-binding residues (9): 226; 11; 69; 69; 138; 142; 143; 144; 204

Post-translational modifications (2): 172, 436

Function

Pathways and Gene Ontology

Reactome pathways

93 pathways

ID	Pathway
R-HSA-1445148	Translocation of SLC2A4 (GLUT4) to the plasma membrane
R-HSA-190840	Microtubule-dependent trafficking of connexons from Golgi to the plasma membrane
R-HSA-190861	Gap junction assembly
R-HSA-2132295	MHC class II antigen presentation
R-HSA-2467813	Separation of Sister Chromatids
R-HSA-2500257	Resolution of Sister Chromatid Cohesion
R-HSA-2565942	Regulation of PLK1 Activity at G2/M Transition
R-HSA-3371497	HSP90 chaperone cycle for steroid hormone receptors (SHR) in the presence of ligand
R-HSA-380259	Loss of Nlp from mitotic centrosomes
R-HSA-380270	Recruitment of mitotic centrosome proteins and complexes
R-HSA-380284	Loss of proteins required for interphase microtubule organization from the centrosome
R-HSA-380320	Recruitment of NuMA to mitotic centrosomes
R-HSA-389957	Prefoldin mediated transfer of substrate to CCT/TriC
R-HSA-389960	Formation of tubulin folding intermediates by CCT/TriC
R-HSA-389977	Post-chaperonin tubulin folding pathway
R-HSA-437239	Recycling pathway of L1
R-HSA-5610787	Hedgehog ‘off’ state
R-HSA-5617833	Cilium Assembly
R-HSA-5620912	Anchoring of the basal body to the plasma membrane
R-HSA-5620924	Intraflagellar transport
R-HSA-5626467	RHO GTPases activate IQGAPs
R-HSA-5663220	RHO GTPases Activate Formins
R-HSA-6807878	COPI-mediated anterograde transport
R-HSA-6811434	COPI-dependent Golgi-to-ER retrograde traffic
R-HSA-6811436	COPI-independent Golgi-to-ER retrograde traffic
R-HSA-68877	Mitotic Prometaphase
R-HSA-8852276	The role of GTSE1 in G2/M progression after G2 checkpoint
R-HSA-8854518	AURKA Activation by TPX2
R-HSA-8955332	Carboxyterminal post-translational modifications of tubulin
R-HSA-9609690	HCMV Early Events

MSigDB gene sets: 391 (showing top): GOBP_NEGATIVE_REGULATION_OF_PROTEIN_CONTAINING_COMPLEX_ASSEMBLY, WU_APOPTOSIS_BY_CDKN1A_VIA_TP53, AP1_01, GOBP_REGULATION_OF_PROTEIN_POLYMERIZATION, MYOGENIN_Q6, GOBP_REGULATION_OF_MICROTUBULE_BASED_PROCESS, REACTOME_ADAPTIVE_IMMUNE_SYSTEM, REACTOME_CYTOKINE_SIGNALING_IN_IMMUNE_SYSTEM, ENK_UV_RESPONSE_KERATINOCYTE_UP, GCANCTGNY_MYOD_Q6, DAZARD_UV_RESPONSE_CLUSTER_G4, GOBP_NEGATIVE_REGULATION_OF_PROTEIN_POLYMERIZATION, TGACCTY_ERR1_Q2, REACTOME_MEMBRANE_TRAFFICKING, CHX10_01

GO Biological Process (5): microtubule cytoskeleton organization (GO:0000226), mitotic cell cycle (GO:0000278), negative regulation of microtubule polymerization (GO:0031115), cytoskeleton organization (GO:0007010), microtubule-based process (GO:0007017)

GO Molecular Function (7): GTPase activity (GO:0003924), structural constituent of cytoskeleton (GO:0005200), calcium ion binding (GO:0005509), GTP binding (GO:0005525), nucleotide binding (GO:0000166), protein binding (GO:0005515), metal ion binding (GO:0046872)

GO Cellular Component (15): nucleus (GO:0005634), cytoplasm (GO:0005737), cytosol (GO:0005829), microtubule (GO:0005874), axoneme (GO:0005930), microtubule cytoskeleton (GO:0015630), internode region of axon (GO:0033269), neuronal cell body (GO:0043025), myelin sheath (GO:0043209), intercellular bridge (GO:0045171), extracellular exosome (GO:0070062), mitotic spindle (GO:0072686), cytoskeleton (GO:0005856), cilium (GO:0005929), cell projection (GO:0042995)

Reactome top-level categories

Rollup of top-16 pathways:

Category	Pathways
Mitotic Prometaphase	2
Centrosome maturation	2
Cooperation of Prefoldin and TriC/CCT in actin and tubulin folding	2
Assembly of the 9+0 primary cilium	2
Membrane Trafficking	1
Transport of connexons to the plasma membrane	1
Gap junction trafficking	1
Adaptive Immune System	1
Mitotic Anaphase	1
G2/M Transition	1
Cellular responses to stress	1
Loss of proteins required for interphase microtubule organization from the centrosome	1
Protein folding	1
L1CAM interactions	1
Signaling by Hedgehog	1

GO top-level categories

Rollup of top GO terms by namespace:

Category	Terms
cellular anatomical structure	7
cytoskeleton	3
cytoskeleton organization	2
microtubule-based process	1
cell cycle	1
mitotic nuclear division	1
negative regulation of microtubule polymerization or depolymerization	1
regulation of microtubule polymerization	1
negative regulation of protein polymerization	1
microtubule polymerization	1
negative regulation of supramolecular fiber organization	1
organelle organization	1
cellular process	1
ribonucleoside triphosphate phosphatase activity	1
structural molecule activity	1
metal ion binding	1
guanyl ribonucleotide binding	1
purine ribonucleoside triphosphate binding	1
nucleoside phosphate binding	1
heterocyclic compound binding	1
binding	1
cation binding	1
intracellular membrane-bounded organelle	1
intracellular anatomical structure	1
cytoplasm	1
microtubule cytoskeleton	1
polymeric cytoskeletal fiber	1
microtubule	1
ciliary plasm	1
main axon	1
somatodendritic compartment	1
cell body	1
extracellular vesicle	1
spindle	1
intracellular membraneless organelle	1
intraciliary transport particle	1
membrane-bounded organelle	1
plasma membrane bounded cell projection	1

Protein interactions and networks

STRING

4574 interactions, top by confidence (×1000):

Protein A	Protein B	Partner UniProt	Score
TUBB4A	TOR1A	O14656	895
TUBB4A	TUBA1B	P04687	860
TUBB4A	TUBA1C	Q9BQE3	860
TUBB4A	TUBA4A	P05215	819
TUBB4A	TUBAL3	A6NHL2	809
TUBB4A	TUBA3E	Q6PEY2	809
TUBB4A	TUBA8	Q9NY65	809
TUBB4A	TUBA1B	P04687	808
TUBB4A	TUBA3C	P0DPH7	808
TUBB4A	THAP1	Q9NVV9	670
TUBB4A	POTEF	A5A3E0	650
TUBB4A	ACTB	P02570	643
TUBB4A	GNAL	P38405	618
TUBB4A	ANO3	Q9BYT9	597
TUBB4A	CIZ1	Q9ULV3	596

IntAct

225 interactions, top by confidence:

A	B	Type	Score
CFTR	ESYT2	psi-mi:“MI:2364”(proximity)	0.710
TUBB	PLD2	psi-mi:“MI:0914”(association)	0.640
MAPK7	PFDN6	psi-mi:“MI:0914”(association)	0.640
RAF1	CALU	psi-mi:“MI:0914”(association)	0.640
TJP1	ACTN4	psi-mi:“MI:0914”(association)	0.600
ILK	HAX1	psi-mi:“MI:0914”(association)	0.530
NEURL4	APBB1	psi-mi:“MI:0914”(association)	0.530
MAPT	KIF2A	psi-mi:“MI:0914”(association)	0.530
CCNJL	PIK3C2A	psi-mi:“MI:0914”(association)	0.530
LRP1	NME4	psi-mi:“MI:0914”(association)	0.530
TSPYL6	NME4	psi-mi:“MI:0914”(association)	0.530
ARL2	TUBB4A	psi-mi:“MI:0914”(association)	0.530
CCT6A	TXNDC9	psi-mi:“MI:0914”(association)	0.530
TUBB2A	EML2	psi-mi:“MI:0914”(association)	0.530
TPSB2	TUBB4A	psi-mi:“MI:0914”(association)	0.530
FAM174A	BLTP3B	psi-mi:“MI:0914”(association)	0.530
TUBB3	POTEF	psi-mi:“MI:0914”(association)	0.530
P/V	IRS4	psi-mi:“MI:0914”(association)	0.530
NR4A1	TUBB4A	psi-mi:“MI:0915”(physical association)	0.520
FLT4	ILVBL	psi-mi:“MI:0914”(association)	0.420
TUBB4A	RPS6KB1	psi-mi:“MI:0915”(physical association)	0.400
SELENOF	TUBB4A	psi-mi:“MI:0915”(physical association)	0.370
TUBB4A	SPG11	psi-mi:“MI:0915”(physical association)	0.370
OTUB1	EPM2A	psi-mi:“MI:0914”(association)	0.350
Ruvbl1	AAR2	psi-mi:“MI:0914”(association)	0.350
TINF2	SIRT2	psi-mi:“MI:0914”(association)	0.350
NFKB1	NFKB1	psi-mi:“MI:0914”(association)	0.350

BioGRID (339): TUBB4A (Affinity Capture-Western), TUBB4A (Affinity Capture-MS), TUBB4A (Affinity Capture-MS), TUBB4A (Affinity Capture-MS), TUBB4A (Affinity Capture-MS), TUBB4A (Affinity Capture-MS), TUBB4A (Affinity Capture-MS), TUBA1C (Co-fractionation), TUBA3E (Co-fractionation), TUBA4A (Co-fractionation), TUBB4A (Two-hybrid), TUBB4A (Proximity Label-MS), TUBB4A (Affinity Capture-MS), TUBB4A (Affinity Capture-MS), TUBB4A (Affinity Capture-MS)

ESM2 similar proteins: A6NNZ2, P02554, P04350, P07437, P09206, P09244, P09652, P11833, P11857, P14643, P18025, P30156, P61857, P61858, P68371, P69893, P69895, P69897, P83130, P99024, Q08115, Q13509, Q24560, Q27U48, Q2HJ81, Q2KJD0, Q2T9S0, Q3MHM5, Q3ZBU7, Q3ZCM7, Q4QRB4, Q4R4X8, Q5R943, Q60HC2, Q6GLE7, Q6P9T8, Q6VAF4, Q767L7, Q7JJU6, Q7KQL5

Diamond homologs: A2AQ07, A6NNZ2, O04386, O17449, O44388, P02554, P04350, P04690, P07436, P07437, P08841, P09203, P09206, P09207, P09244, P09652, P09653, P10876, P10878, P11482, P11833, P11857, P12456, P13602, P14643, P18241, P20365, P22852, P30883, P32882, P33188, P34108, P36221, P37832, P41352, P41387, P41937, P46265, P50261, P50262

SIGNOR signaling

2 interactions.

A	Effect	B	Mechanism
NUMA1	up-regulates	TUBB4A	binding
“Vincristine sulfate”	“down-regulates activity”	TUBB4A	“chemical inhibition”

Enriched among interaction partners

Reactome pathways and GO biological processes over-represented among this gene’s 244 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:

Pathway	Partners	Fold	FDR
RIP-mediated NFkB activation via ZBP1	9	36.6×	8e-11
Formation of tubulin folding intermediates by CCT/TriC	11	28.2×	2e-11
TRAF6 mediated NF-kB activation	10	27.7×	9e-11
Cooperation of Prefoldin and TriC/CCT in actin and tubulin folding	11	27.2×	2e-11
TNF signaling	9	23.1×	7e-09
Post-chaperonin tubulin folding pathway	8	23.1×	5e-08
Microtubule-dependent trafficking of connexons from Golgi to the plasma membrane	7	23.1×	4e-07
Transport of connexons to the plasma membrane	7	23.1×	4e-07

GO biological processes:

GO term	Partners	Fold	FDR
non-canonical NF-kappaB signal transduction	13	51.2×	9e-18
canonical NF-kappaB signal transduction	17	29.1×	6e-18
tumor necrosis factor-mediated signaling pathway	14	21.6×	9e-13
interleukin-1-mediated signaling pathway	5	18.8×	6e-04
response to muscle stretch	5	17.9×	8e-04
toll-like receptor 4 signaling pathway	7	17.2×	3e-05
cytoplasmic microtubule organization	7	11.2×	4e-04
obsolete positive regulation of NF-kappaB transcription factor activity	10	9.6×	3e-05

Disease & clinical

Clinical variants and AI predictions

ClinVar

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

Classification	Count (floor)
Pathogenic	8
Likely pathogenic	24
Uncertain significance	132
Likely benign	117
Benign	25

Top pathogenic / likely-pathogenic (30)

Variant ID	HGVS	Classification
267773	NM_006087.4(TUBB4A):c.4C>T (p.Arg2Trp)	Pathogenic
267777	NM_006087.4(TUBB4A):c.731G>T (p.Gly244Val)	Pathogenic
267781	NM_006087.3:c.900C>A	Pathogenic
267792	NM_006087.4(TUBB4A):c.1164G>A (p.Met388Ile)	Pathogenic
2843042	NM_006087.4(TUBB4A):c.755A>G (p.Lys252Arg)	Pathogenic
372789	NM_006087.4(TUBB4A):c.535G>C (p.Val179Leu)	Pathogenic
3769850	NM_006087.4(TUBB4A):c.1164G>T (p.Met388Ile)	Pathogenic
803516	NM_006087.4(TUBB4A):c.730G>C (p.Gly244Arg)	Pathogenic
1033070	NM_006087.4(TUBB4A):c.854C>A (p.Thr285Lys)	Likely pathogenic
1256060	NM_006087.4(TUBB4A):c.796T>A (p.Phe266Ile)	Likely pathogenic
139453	NM_006087.4(TUBB4A):c.533C>G (p.Thr178Arg)	Likely pathogenic
1450009	NM_006087.4(TUBB4A):c.736C>A (p.Leu246Met)	Likely pathogenic
1694453	NM_006087.4(TUBB4A):c.544C>T (p.Pro182Ser)	Likely pathogenic
1805115	NM_006087.4(TUBB4A):c.493A>G (p.Asn165Asp)	Likely pathogenic
2442020	NM_006087.4(TUBB4A):c.211G>A (p.Gly71Ser)	Likely pathogenic
267780	NM_006087.4(TUBB4A):c.874C>A (p.Gln292Lys)	Likely pathogenic
267789	NM_006087.4(TUBB4A):c.1099T>C (p.Phe367Leu)	Likely pathogenic
267790	NM_006087.4(TUBB4A):c.1162A>G (p.Met388Val)	Likely pathogenic
3336788	NM_006087.4(TUBB4A):c.1052C>T (p.Thr351Met)	Likely pathogenic
3600322	NM_006087.4(TUBB4A):c.287G>T (p.Gly96Val)	Likely pathogenic
453295	NM_006087.4(TUBB4A):c.1062C>G (p.Cys354Trp)	Likely pathogenic
4533373	NM_006087.4(TUBB4A):c.898A>G (p.Met300Val)	Likely pathogenic
4813348	NM_006087.4(TUBB4A):c.937G>T (p.Val313Leu)	Likely pathogenic
4819874	NM_006087.4(TUBB4A):c.784C>T (p.Arg262Cys)	Likely pathogenic
580978	NM_006087.4(TUBB4A):c.686T>C (p.Val229Ala)	Likely pathogenic
689794	NM_006087.4(TUBB4A):c.1181T>C (p.Phe394Ser)	Likely pathogenic
807518	NM_006087.4(TUBB4A):c.1065C>A (p.Asp355Glu)	Likely pathogenic
807519	NM_006087.4(TUBB4A):c.1054G>T (p.Ala352Ser)	Likely pathogenic
871906	NM_006087.4(TUBB4A):c.1049A>C (p.Lys350Thr)	Likely pathogenic
930298	NM_006087.4(TUBB4A):c.1021T>C (p.Phe341Leu)	Likely pathogenic

SpliceAI

533 predictions. Top by Δscore:

Variant	Effect	Δscore
19:6496217:TTGGC:T	acceptor_gain	1.0000
19:6496218:TGGC:T	acceptor_gain	1.0000
19:6496219:GGC:G	acceptor_gain	1.0000
19:6496220:GC:G	acceptor_gain	1.0000
19:6496221:CC:C	acceptor_gain	1.0000
19:6496221:CCTA:C	acceptor_loss	1.0000
19:6496222:C:CC	acceptor_gain	1.0000
19:6496222:C:G	acceptor_loss	1.0000
19:6496223:T:G	acceptor_loss	1.0000
19:6501281:ACTC:A	donor_loss	1.0000
19:6501282:CTCA:C	donor_loss	1.0000
19:6501283:TCACC:T	donor_loss	1.0000
19:6501284:CAC:C	donor_loss	1.0000
19:6501285:A:AC	donor_gain	1.0000
19:6501285:A:C	donor_loss	1.0000
19:6501286:C:CC	donor_gain	1.0000
19:6501286:C:T	donor_loss	1.0000
19:6501393:TCCTC:T	acceptor_gain	1.0000
19:6501394:CCTC:C	acceptor_gain	1.0000
19:6501394:CCTCC:C	acceptor_gain	1.0000
19:6501395:CTC:C	acceptor_gain	1.0000
19:6501395:CTCC:C	acceptor_gain	1.0000
19:6501396:TC:T	acceptor_gain	1.0000
19:6501396:TCCT:T	acceptor_gain	1.0000
19:6501397:CC:C	acceptor_gain	1.0000
19:6501398:C:CC	acceptor_gain	1.0000
19:6501398:CTG:C	acceptor_loss	1.0000
19:6501401:C:CT	acceptor_gain	1.0000
19:6501510:CCTA:C	donor_loss	1.0000
19:6501511:CTAC:C	donor_loss	1.0000

AlphaMissense

2954 scored. Top likely-pathogenic:

Variant	Protein change	am_pathogenicity
19:6495235:A:G	Y422H	1.000
19:6495246:A:G	L418P	1.000
19:6495249:T:A	D417V	1.000
19:6495250:C:G	D417H	1.000
19:6495268:C:G	A411P	1.000
19:6495275:G:C	F408L	1.000
19:6495275:G:T	F408L	1.000
19:6495276:A:G	F408S	1.000
19:6495277:A:G	F408L	1.000
19:6495291:A:G	M403T	1.000
19:6495295:C:G	G402R	1.000
19:6495310:A:G	W397R	1.000
19:6495310:A:T	W397R	1.000
19:6495313:G:C	H396D	1.000
19:6495317:G:C	F394L	1.000
19:6495317:G:T	F394L	1.000
19:6495318:A:G	F394S	1.000
19:6495319:A:G	F394L	1.000
19:6495319:A:T	F394I	1.000
19:6495344:G:C	F385L	1.000
19:6495344:G:T	F385L	1.000
19:6495346:A:G	F385L	1.000
19:6495389:G:C	N370K	1.000
19:6495389:G:T	N370K	1.000
19:6495465:A:G	I345T	1.000
19:6495467:C:A	W344C	1.000
19:6495467:C:G	W344C	1.000
19:6495469:A:G	W344R	1.000
19:6495469:A:T	W344R	1.000
19:6495567:A:G	L311P	1.000

dbSNP variants (sampled 300 via entrez): RS1000087027 (19:6494506 G>C), RS1000647524 (19:6496492 G>A), RS1000767321 (19:6504493 G>T), RS1001178554 (19:6495362 C>G,T), RS1002189307 (19:6494357 A>G,T), RS1002198900 (19:6499894 C>G), RS1002301585 (19:6500001 G>T), RS1002698276 (19:6500111 G>A), RS1003146368 (19:6498842 T>C), RS1003362960 (19:6504679 A>G,T), RS1004162746 (19:6497253 C>A,T), RS1004382616 (19:6496660 T>A), RS1004531669 (19:6498053 T>C), RS1004630955 (19:6504107 G>A,C), RS1004776318 (19:6503219 C>A,G,T)

Disease associations

OMIM: gene MIM:602662 | disease phenotypes: MIM:612438, MIM:128101, MIM:312080, MIM:616407, MIM:303350

GenCC curated gene-disease

Disease	Classification	Inheritance
hypomyelinating leukodystrophy 6	Definitive	Autosomal dominant
torsion dystonia 4	Strong	Autosomal dominant
TUBB4A-related neurologic disorder	Moderate	Autosomal dominant

ClinGen Gene-Disease Validity (1)

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

Disease	Classification	Inheritance
TUBB4A-related neurologic disorder	Definitive	AD

Mondo (12): hypomyelinating leukodystrophy 6 (MONDO:0012905), torsion dystonia 4 (MONDO:0007493), cerebral palsy (MONDO:0006497), classic medulloblastoma (MONDO:0016712), microcephaly (MONDO:0001149), leukodystrophy (MONDO:0019046), TUBB4A-related neurologic disorder (MONDO:0800470), Brown syndrome (MONDO:0014624), frontotemporal dementia (MONDO:0017276), intellectual disability (MONDO:0001071), hereditary spastic paraplegia (MONDO:0019064), congenital nervous system disorder (MONDO:0002320)

Orphanet (7): Hypomyelination with atrophy of basal ganglia and cerebellum (Orphanet:139441), Primary dystonia, DYT4 type (Orphanet:98805), Classic medulloblastoma (Orphanet:251867), Leukodystrophy (Orphanet:68356), Frontotemporal dementia (Orphanet:282), Hereditary spastic paraplegia (Orphanet:685), NON RARE IN EUROPE: Unexplained intellectual disability (Orphanet:319658)

HPO phenotypes

56 total (30 of 56 shown, HPO-id order):

HPO	Term
HP:0000006	Autosomal dominant inheritance
HP:0000182	Movement abnormality of the tongue
HP:0000194	Open mouth
HP:0000252	Microcephaly
HP:0000275	Narrow face
HP:0000473	Torticollis
HP:0000505	Visual impairment
HP:0000571	Hypometric saccades
HP:0000639	Nystagmus
HP:0000643	Blepharospasm
HP:0000648	Optic atrophy
HP:0000657	Oculomotor apraxia
HP:0000726	Dementia
HP:0000750	Delayed speech and language development
HP:0001249	Intellectual disability
HP:0001250	Seizure
HP:0001251	Ataxia
HP:0001252	Hypotonia
HP:0001257	Spasticity
HP:0001260	Dysarthria
HP:0001266	Choreoathetosis
HP:0001270	Motor delay
HP:0001272	Cerebellar atrophy
HP:0001288	Gait disturbance
HP:0001304	Torsion dystonia
HP:0001328	Specific learning disability
HP:0001332	Dystonia
HP:0001337	Tremor
HP:0001533	Slender build
HP:0001618	Dysphonia

GWAS associations

2 associations (top):

Study	Trait	p-value
GCST90010427_25	Left–right brain asymmetry	6.000000e-10
GCST90013421_20	Left-handedness	6.000000e-12

EFO canonical traits (1, from GWAS)

EFO ID	Trait name
EFO:0009902	handedness

MeSH disease descriptors (6)

Descriptor	Name	Tree numbers
D002547	Cerebral Palsy	C10.228.140.140.254
D057180	Frontotemporal Dementia	C10.228.140.380.266.299; C10.574.950.300.299; C18.452.845.800.300.299; F03.615.400.380.299
D008607	Intellectual Disability	C10.597.606.360; C23.888.592.604.646; F01.700.687; F03.625.539
D008831	Microcephaly	C05.660.207.620; C10.500.507.400.500; C16.131.621.207.620; C16.131.666.507.400.500
D015419	Spastic Paraplegia, Hereditary	C10.500.300.820; C10.574.500.495.820; C10.668.829.800.300.820; C16.131.666.300.820; C16.320.400.375.820
C567314	Leukodystrophy, Hypomyelinating, 6 (supp.)

Drugs & pharmacology

Drug and pharmacology data

Is drug target: yes

ChEMBL targets (4): CHEMBL2095182 (PROTEIN COMPLEX GROUP), CHEMBL3832942 (PROTEIN FAMILY), CHEMBL3838 (SINGLE PROTEIN), CHEMBL6066847 (PROTEIN-PROTEIN INTERACTION)

Molecules with ChEMBL bioactivity

21 molecules (phase ≥1), by development phase (incl. off-target/promiscuous compounds). Patent mentions across the top 20 by phase: 1,641,397 (via chembl_molecule»patent_compound — counts attach to the compound, not the gene–compound relationship, so off-target/promiscuous molecules can dominate).

Molecule	Name	Phase	Patents
CHEMBL107	COLCHICINE	4	93,932
CHEMBL159	VINBLASTINE	4	412,636
CHEMBL33	LEVOFLOXACIN ANHYDROUS	4	43,403
CHEMBL3545252	DOCETAXEL	4	1,009
CHEMBL364713	NOSCAPINE	4	14,987
CHEMBL378544	VINBLASTINE SULFATE	4	32,829
CHEMBL428647	PACLITAXEL	4	332,542
CHEMBL5315124	LEVOFLOXACIN	4	189
CHEMBL553025	VINORELBINE	4	142,159
CHEMBL571546	TIRBANIBULIN	4	1,192
CHEMBL61	PODOFILOX	4	37,640
CHEMBL90555	VINCRISTINE	4	268,031
CHEMBL92	DOCETAXEL ANHYDROUS	4	196,686
CHEMBL94657	PATUPILONE	3	14,934
CHEMBL20684	ABT-751	2	2,238
CHEMBL292702	MAYTANSINE	2	9,300
CHEMBL39541	DOLASTATIN-10	2	1,380
CHEMBL49642	INDIBULIN	2	963
CHEMBL528271	PARBENDAZOLE	2	6,102
CHEMBL9514	NOCODAZOLE	2	29,245
CHEMBL246600	COMBRETASTATIN	1

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

ChEMBL bioactivities

1170 potent at pChembl≥5 of 1321 total, top 50 by pChembl (potency: 10 = 0.1 nM, 6 = 1 µM).

pChembl	Type	Value	Unit	Molecule
10.26	IC50	0.0551	nM	CHEMBL5194719
9.75	IC50	0.176	nM	CHEMBL5203711
9.51	IC50	0.308	nM	VINORELBINE
9.30	IC50	0.5	nM	DOLASTATIN-10
8.92	IC50	1.2	nM	CHEMBL5169466
8.85	IC50	1.4	nM	CHEMBL4575066
8.74	IC50	1.8	nM	PATUPILONE
8.59	IC50	2.6	nM	CHEMBL2024544
8.52	Ki	3	nM	COMBRETASTATIN A4
8.52	IC50	3	nM	COMBRETASTATIN A4
8.52	IC50	3	nM	DOLASTATIN-10
8.52	Kd	3	nM	CHEMBL5410715
8.52	IC50	3	nM	COLCHICINE
8.46	IC50	3.5	nM	PATUPILONE
8.38	IC50	4.2	nM	CHEMBL5197739
8.27	IC50	5.4	nM	CHEMBL5280519
8.22	IC50	6	nM	CHEMBL498271
8.20	Kd	6.342	nM	CHEMBL3752910
8.20	ED50	6.342	nM	CHEMBL3752910
8.05	EC50	9	nM	COMBRETASTATIN A4
8.01	EC50	9.8	nM	COMBRETASTATIN A4
8.01	IC50	9.77	nM	CHEMBL4778826
8.00	Ki	10	nM	CHEMBL381852
8.00	IC50	10	nM	CHEMBL204241
7.96	Kd	11	nM	CHEMBL5611905
7.89	IC50	12.9	nM	COMBRETASTATIN A4
7.85	Kd	14	nM	MAYTANSINE
7.70	IC50	20	nM	CHEMBL2369093
7.70	Ki	20	nM	CHEMBL369927
7.70	IC50	20	nM	CHEMBL5183440
7.70	Kd	20	nM	CHEMBL5613121
7.67	Kd	21.5	nM	CHEMBL4797381
7.64	IC50	23	nM	COMBRETASTATIN A4
7.62	EC50	24	nM	CHEMBL4250191
7.60	IC50	25	nM	CHEMBL552462
7.52	IC50	30	nM	CHEMBL381122
7.52	IC50	30	nM	COMBRETASTATIN A4
7.52	IC50	30	nM	CHEMBL203062
7.52	IC50	30	nM	CHEMBL204710
7.51	EC50	31	nM	PACLITAXEL
7.43	Kd	36.9	nM	CHEMBL4756812
7.40	EC50	40	nM	CHEMBL1688184
7.35	EC50	45	nM	CHEMBL4239716
7.34	IC50	46	nM	CHEMBL374257
7.33	Kd	47	nM	CHEMBL5542101
7.30	Ki	50	nM	CHEMBL196966
7.30	EC50	50	nM	CHEMBL1688183
7.30	EC50	50	nM	CHEMBL1688189
7.29	Kd	51	nM	CHEMBL5614306
7.28	EC50	53	nM	CHEMBL4241638

PubChem BioAssay actives

1098 with measured affinity, of 5890 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.

Compound	Assay	Type	Value	Unit
(Z)-1-[4-bromo-2-(4-fluorophenyl)-1-benzofuran-7-yl]-3-(4-methoxyphenyl)prop-2-en-1-one	1882651: Inhibition of tubulin polymerization (unknown origin) measured every 3 secs for 1 hr by spectroflourimetric analysis	ic50	0.0001	uM
(Z)-1-[4-bromo-2-(4-fluorophenyl)-1-benzofuran-7-yl]-3-[4-(trifluoromethoxy)phenyl]prop-2-en-1-one	1882651: Inhibition of tubulin polymerization (unknown origin) measured every 3 secs for 1 hr by spectroflourimetric analysis	ic50	0.0002	uM
Vinorelbine	1993632: Inhibition of tubulin polymerization (unknown origin)	ic50	0.0003	uM
(2S)-2-[[(2S)-2-(dimethylamino)-3-methylbutanoyl]amino]-N-[(3R,4S,5S)-3-methoxy-1-[(2S)-2-[(1R,2R)-1-methoxy-2-methyl-3-oxo-3-[[(1S)-2-phenyl-1-(1,3-thiazol-2-yl)ethyl]amino]propyl]pyrrolidin-1-yl]-5-methyl-1-oxoheptan-4-yl]-N,3-dimethylbutanamide	270819: Inhibition of tubulin polymerization	ic50	0.0005	uM
(2S)-2-[[(2S)-2-(dimethylamino)-3-methylbutanoyl]amino]-N-[(3R,4S,5S)-3-methoxy-1-[(2S)-2-[(1R,2R)-1-methoxy-2-methyl-3-oxo-3-(2-pyridin-2-ylethylamino)propyl]pyrrolidin-1-yl]-5-methyl-1-oxoheptan-4-yl]-N,3-dimethylbutanamide	1896115: Binding affinity to tubulin (unknown origin)	ic50	0.0012	uM
(3Z,6Z)-3-[(5-tert-butyl-1H-imidazol-4-yl)methylidene]-6-phenacylidenepiperazine-2,5-dione	1587857: Inhibition of tubulin polymerization (unknown origin)	ic50	0.0014	uM
(3Z,6Z)-3-[(5-tert-butyl-1H-imidazol-4-yl)methylidene]-6-[(2,5-difluorophenyl)methylidene]piperazine-2,5-dione	1587857: Inhibition of tubulin polymerization (unknown origin)	ic50	0.0026	uM
3-hydroxy-2-[N-[2-(methoxymethyl)-1-benzofuran-7-yl]-C-methylcarbonimidoyl]-5-phenylcyclohex-2-en-1-one	2034736: Displacement of fluorescent probe (R)-(+)-ethyl 5-amino2-methyl-1,2-dihydro-3-phenylpyrido[3,4-b]pyrazin-7-ylcarbamate from tubulin colchicine binding site (unknown origin) assessed as dissociation constant	kd	0.0030	uM
2-methoxy-5-[(Z)-2-(3,4,5-trimethoxyphenyl)ethenyl]phenol	1862628: Inhibition of tubulin polymerization in human HeLa cells assessed as microtubule network polymerization after 30 mins by immunofluorescence analysis	ic50	0.0030	uM
Colchicine	2074087: Inhibition of microtubule polymerization in human K562 cells measured for 60 mins by fluorescence based analysis	ic50	0.0030	uM
(1S,3S,7S,10R,11S,12S,16R)-7,11-dihydroxy-8,8,10,12,16-pentamethyl-3-[(E)-1-(2-methyl-1,3-thiazol-4-yl)prop-1-en-2-yl]-4,17-dioxabicyclo[14.1.0]heptadecane-5,9-dione	1408257: Inhibition of tubulin polymerization in human MCF7 cells assessed as induction of mitotic arrest	ic50	0.0035	uM
tert-butyl (3R,4S,5S)-4-[[(2S)-2-[[(2S)-2-(dimethylamino)-3-methylbutanoyl]amino]-3-methylbutanoyl]-methylamino]-3-methoxy-5-methylheptanoate	1896110: Inhibition of tubulin (unknown origin) polymerization assessed as reduction in microtubule formation measured for 20 mins by spectrophotometric analysis	ic50	0.0042	uM
3-methoxy-6-[4-(3,4,5-trimethoxyphenyl)-1H-pyrazol-5-yl]benzene-1,2-diol	1929685: Inhibition of tubulin polymerization in human SH-SY5Y cells incubated for 24 hrs by SDS-PAGE based analysis	ic50	0.0054	uM
2-methoxy-5-[1-(3,4,5-trimethoxyphenyl)ethenyl]phenol	1862628: Inhibition of tubulin polymerization in human HeLa cells assessed as microtubule network polymerization after 30 mins by immunofluorescence analysis	ic50	0.0060	uM
4-methyl-3-[(1-methyl-6-pyridin-3-ylpyrazolo[3,4-d]pyrimidin-4-yl)amino]-N-[3-(trifluoromethyl)phenyl]benzamide	2149956: Binding affinity to human TUBB4A incubated for 45 mins by Kinobead based pull down assay	kd	0.0063	uM
(E)-1-[1-(4-chlorophenyl)-5-methyltriazol-4-yl]-3-(3,4-dimethoxyphenyl)prop-2-en-1-one	1689700: Inhibition of Tubulin in human RPMI-8226 cells incubated for 2 hrs by ELISA	ic50	0.0098	uM
N-(1,3-benzodioxol-5-yl)-5-[2-(3,5-dimethoxyphenoxy)-3-pyridinyl]-1H-1,2,4-triazol-3-amine	256882: Displacement of [3H]colchicine from tubulin	ki	0.0100	uM
N-(2,3-dihydro-1,4-benzodioxin-6-yl)-5-[2-(pyridin-2-ylmethylamino)phenyl]-1,3-oxazol-2-amine	261214: Displacement of [3H]colchicine from tubulin at 65 nM	ic50	0.0100	uM
[(1S,2R,3S,5R,6S,16E,18E,20R,21S)-11-chloro-21-hydroxy-12,20-dimethoxy-2,5,9,16-tetramethyl-8,23-dioxo-4,24-dioxa-9,22-diazatetracyclo[19.3.1.110,14.03,5]hexacosa-10,12,14(26),16,18-pentaen-6-yl] acetate	2126113: Displacement of fluorescent Maytansine probe from tubulin (unknown origin) assessed as dissociation constant by competitive binding based fluorescence anisotropy	kd	0.0110	uM
[(1S,2R,3S,5S,6S,16E,18E,20R,21S)-11-chloro-21-hydroxy-12,20-dimethoxy-2,5,9,16-tetramethyl-8,23-dioxo-4,24-dioxa-9,22-diazatetracyclo[19.3.1.110,14.03,5]hexacosa-10,12,14(26),16,18-pentaen-6-yl] (2S)-2-[acetyl(methyl)amino]propanoate	2126113: Displacement of fluorescent Maytansine probe from tubulin (unknown origin) assessed as dissociation constant by competitive binding based fluorescence anisotropy	kd	0.0140	uM
N-(2,3-dihydro-1,4-benzodioxin-6-yl)-5-[2-(3,5-dimethoxyphenoxy)-3-pyridinyl]-1H-1,2,4-triazol-3-amine	256882: Displacement of [3H]colchicine from tubulin	ki	0.0200	uM
methyl (13S,15S,17S)-13-[(1R,9R,10S,11R,12R,19R)-10-(1-diethoxyphosphorylhexylcarbamoyl)-12-ethyl-10,11-dihydroxy-5-methoxy-8-methyl-8,16-diazapentacyclo[10.6.1.01,9.02,7.016,19]nonadeca-2,4,6,13-tetraen-4-yl]-17-ethyl-17-hydroxy-1,11-diazatetracyclo[13.3.1.04,12.05,10]nonadeca-4(12),5,7,9-tetraene-13-carboxylate	214333: The compound tested for the inhibition of tubulin polymerization.	ic50	0.0200	uM
(E)-3-[5-[(2-cyanoquinolin-4-yl)-methylamino]-2-methoxyphenyl]-N-hydroxyprop-2-enamide	1862628: Inhibition of tubulin polymerization in human HeLa cells assessed as microtubule network polymerization after 30 mins by immunofluorescence analysis	ic50	0.0200	uM
[(1S,2R,3S,5R,6S,16E,18E,20R,21S)-11-chloro-21-hydroxy-12,20-dimethoxy-2,5,9,16-tetramethyl-8,23-dioxo-4,24-dioxa-9,22-diazatetracyclo[19.3.1.110,14.03,5]hexacosa-10,12,14(26),16,18-pentaen-6-yl] hept-6-ynoate	2126113: Displacement of fluorescent Maytansine probe from tubulin (unknown origin) assessed as dissociation constant by competitive binding based fluorescence anisotropy	kd	0.0200	uM
(3S)-3-[(5R)-6-[[1-(4-fluorophenyl)triazol-4-yl]methyl]-4-methoxy-7,8-dihydro-5H-[1,3]dioxolo[4,5-g]isoquinolin-5-yl]-6,7-dimethoxy-3H-2-benzofuran-1-one	1675128: Binding affinity to CM5 chip immobilized beta tubulin (unknown origin) assessed as thermodynamic constants by SPR assay	kd	0.0215	uM
N-(4-methoxyphenyl)-N,5-dimethylfuro[2,3-d]pyrimidin-4-amine	1635267: Induction of microtubule depolymerization in human A10 cells incubated for 18 hrs by indirect immunofluorescence analysis	ec50	0.0240	uM
[4-amino-2-(4-methoxyanilino)-1,3-thiazol-5-yl]-(3,4-dimethoxyphenyl)methanone	1674862: Binding affinity to beta tubulin in HEK293 cells assessed as disruption of microtubule polymerization by ITDRF-CETSA assay	ic50	0.0250	uM
N-(2,3-dihydro-1,4-benzodioxin-6-yl)-5-[2-(pyridin-3-ylmethylamino)-3-pyridinyl]-1,3,4-oxadiazol-2-amine	261214: Displacement of [3H]colchicine from tubulin at 65 nM	ic50	0.0300	uM
N-(1,3-benzodioxol-5-yl)-5-[2-(pyridin-4-ylmethylamino)-3-pyridinyl]-1,3,4-oxadiazol-2-amine	261214: Displacement of [3H]colchicine from tubulin at 65 nM	ic50	0.0300	uM
N-(1,3-benzodioxol-5-yl)-5-[2-(pyridin-2-ylmethylamino)-3-pyridinyl]-1,3,4-oxadiazol-2-amine	261214: Displacement of [3H]colchicine from tubulin at 65 nM	ic50	0.0300	uM
Paclitaxel	260235: Effect on induction of mitotic arrest by phosphohistone H3 (pH3) assay	ec50	0.0310	uM
(3S)-3-[(5R)-9-bromo-6-[[1-(4-bromophenyl)triazol-4-yl]methyl]-4-methoxy-7,8-dihydro-5H-[1,3]dioxolo[4,5-g]isoquinolin-5-yl]-6,7-dimethoxy-3H-2-benzofuran-1-one	1675128: Binding affinity to CM5 chip immobilized beta tubulin (unknown origin) assessed as thermodynamic constants by SPR assay	kd	0.0369	uM
6-(3-chloro-6,11,11-trioxobenzo[b][1,4]benzothiazepin-5-yl)-N-hydroxyhexanamide	580932: Inhibition of HDAC activity in human NB4 cells assessed as acetylated tubulin after 24 hrs by Western blot analysis	ec50	0.0400	uM
N,5-dimethyl-N-(4-methylsulfanylphenyl)furo[2,3-d]pyrimidin-4-amine	1635267: Induction of microtubule depolymerization in human A10 cells incubated for 18 hrs by indirect immunofluorescence analysis	ec50	0.0450	uM
[7-(3-hydroxyprop-1-ynyl)-6-methoxy-2H-indazol-3-yl]-(3,4,5-trimethoxyphenyl)methanone	280080: Displacement of [3H]colchicine from tubulin in MCF7 cells	ic50	0.0460	uM
(3S,10R,13E,16S)-10-[(3-chloro-4-methoxyphenyl)methyl]-6,6-dimethyl-3-(2-methylpropyl)-16-[(1S)-1-[(2R,3S)-3-phenyloxiran-2-yl]ethyl]-1,4-dioxa-8,11-diazacyclohexadec-13-ene-2,5,9,12-tetrone	2061859: Binding affinity to tubulin (unknown origin) assessed as dissociation constant by SPR analysis	kd	0.0470	uM
N-hydroxy-6-(3-methoxy-6,11,11-trioxobenzo[b][1,4]benzothiazepin-5-yl)hexanamide	580932: Inhibition of HDAC activity in human NB4 cells assessed as acetylated tubulin after 24 hrs by Western blot analysis	ec50	0.0500	uM
N-hydroxy-6-(6,11,11-trioxobenzo[b][1,4]benzothiazepin-5-yl)hexanamide	580932: Inhibition of HDAC activity in human NB4 cells assessed as acetylated tubulin after 24 hrs by Western blot analysis	ec50	0.0500	uM
5-[2-(3,5-dimethoxyphenoxy)-3-pyridinyl]-N-(3-methoxyphenyl)-1H-1,2,4-triazol-3-amine	256882: Displacement of [3H]colchicine from tubulin	ki	0.0500	uM
[(1S,2R,3S,5R,6S,16E,18E,20R,21S)-11-chloro-21-hydroxy-12,20-dimethoxy-2,5,9,16-tetramethyl-8,23-dioxo-4,24-dioxa-9,22-diazatetracyclo[19.3.1.110,14.03,5]hexacosa-10,12,14(26),16,18-pentaen-6-yl] benzoate	2126113: Displacement of fluorescent Maytansine probe from tubulin (unknown origin) assessed as dissociation constant by competitive binding based fluorescence anisotropy	kd	0.0510	uM
N-ethyl-N-(4-methoxyphenyl)-5-methylfuro[2,3-d]pyrimidin-4-amine	1635267: Induction of microtubule depolymerization in human A10 cells incubated for 18 hrs by indirect immunofluorescence analysis	ec50	0.0530	uM
3-[3-(1,3-benzodioxol-5-ylamino)-1H-1,2,4-triazol-5-yl]-N-(3,5-dimethoxyphenyl)pyridin-2-amine	256882: Displacement of [3H]colchicine from tubulin	ki	0.0600	uM
Vinblastine	214333: The compound tested for the inhibition of tubulin polymerization.	ic50	0.0700	uM
methyl (13S,15S,17S)-13-[(1R,9R,10S,11R,12R,19R)-10-[(1-diethoxyphosphoryl-2-phenylethyl)carbamoyl]-12-ethyl-10,11-dihydroxy-5-methoxy-8-methyl-8,16-diazapentacyclo[10.6.1.01,9.02,7.016,19]nonadeca-2,4,6,13-tetraen-4-yl]-17-ethyl-17-hydroxy-1,11-diazatetracyclo[13.3.1.04,12.05,10]nonadeca-4(12),5,7,9-tetraene-13-carboxylate	214333: The compound tested for the inhibition of tubulin polymerization.	ic50	0.0700	uM
N-(2,3-dihydro-1,4-benzodioxin-6-yl)-5-[3-(pyridin-2-ylmethylamino)thiophen-2-yl]-1,3,4-oxadiazol-2-amine	261214: Displacement of [3H]colchicine from tubulin at 65 nM	ic50	0.0750	uM
methyl (13S,15S,17S)-13-[(1R,9R,10S,11R,12R,19R)-10-[[(1S)-1-diethoxyphosphorylethyl]carbamoyl]-12-ethyl-10,11-dihydroxy-5-methoxy-8-methyl-8,16-diazapentacyclo[10.6.1.01,9.02,7.016,19]nonadeca-2,4,6,13-tetraen-4-yl]-17-ethyl-17-hydroxy-1,11-diazatetracyclo[13.3.1.04,12.05,10]nonadeca-4(12),5,7,9-tetraene-13-carboxylate	214333: The compound tested for the inhibition of tubulin polymerization.	ic50	0.0800	uM
4-methoxy-2-[(Z)-2-(3,4,5-trimethoxyphenyl)ethenyl]thiophene	1267532: Inhibition of Tubulin polymerization in human HeLa cells assessed as decrease in dynamic tyrosinated microtubules after 2 hrs by microplate reader analysis	ec50	0.0810	uM
N-[2-[[(E)-(2,4-dihydroxyphenyl)methylideneamino]carbamoyl]phenyl]furan-2-carboxamide	1882654: Inhibition of tubulin polymerization (unknown origin)	ic50	0.0876	uM
N-hydroxy-6-(3-methoxy-6-oxobenzo[b][1,4]benzoxazepin-5-yl)hexanamide	580932: Inhibition of HDAC activity in human NB4 cells assessed as acetylated tubulin after 24 hrs by Western blot analysis	ec50	0.0900	uM
N-(3,5-dimethoxyphenyl)-3-[3-(3-methoxyanilino)-1H-1,2,4-triazol-5-yl]pyridin-2-amine	256882: Displacement of [3H]colchicine from tubulin	ki	0.1000	uM

CTD chemical–gene interactions

50 total (human), top 30 by PubMed support.

Chemical	Actions (top 5)	PubMed papers
entinostat	increases expression, affects cotreatment	2
4-(5-benzo(1,3)dioxol-5-yl-4-pyridin-2-yl-1H-imidazol-2-yl)benzamide	affects cotreatment, increases expression	2
Rotenone	decreases expression	2
Valproic Acid	affects expression, increases methylation	2
aristolochic acid I	increases expression	1
6,7-dimethoxy-2-(pyrrolidin-1-yl)-N-(5-(pyrrolidin-1-yl)pentyl)quinazolin-4-amine	decreases expression	1
FR900359	increases phosphorylation	1
beauvericin	affects cotreatment, increases expression	1
triphenyl phosphate	affects expression	1
ethyl-p-hydroxybenzoate	decreases expression	1
trichostatin A	affects expression	1
arsenite	affects binding, increases reaction	1
sulforaphane	decreases expression	1
11-nor-delta(9)-tetrahydrocannabinol-9-carboxylic acid	affects methylation, increases abundance	1
sodium arsenite	increases expression	1
tobacco tar	decreases expression, decreases reaction	1
diallyl disulfide	decreases expression, decreases reaction	1
S-(1,2-dichlorovinyl)cysteine	affects response to substance, increases expression	1
di-n-butylphosphoric acid	affects expression	1
perfluorooctane sulfonic acid	affects expression	1
enniatins	affects cotreatment, increases expression	1
poly(propyleneimine)	increases expression	1
abrine	decreases expression	1
dorsomorphin	affects cotreatment, increases expression	1
hexabrominated diphenyl ether 153	decreases expression	1
perfluorobutanesulfonic acid	affects expression	1
LDN 193189	affects cotreatment, increases expression	1
4-(4-((5-(4,5-dimethyl-2-nitrophenyl)-2-furanyl)methylene)-4,5-dihydro-3-methyl-5-oxo-1H-pyrazol-1-yl)benzoic acid	decreases expression	1
Resveratrol	affects cotreatment, decreases expression	1
Sunitinib	decreases expression	1

ChEMBL screening assays

1758 unique, capped per target: 1718 binding, 34 functional, 6 admet

Representative assays (with source publication via chembl_document):

Assay ID	Type	Description	Source paper
CHEMBL1009021	Binding	Inhibition of tubulin polymerization in human MCF7 cells at 1 uM after 2 hrs by SDS-PAGE	A new cytotoxic epothilone from modified polyketide synthases heterologously expressed in Myxococcus xanthus. — J Nat Prod
CHEMBL4146506	ADMET	Inhibition of tubulin polymerization in human HaCaT cells assessed as chromatin condensation at 1 uM after 24 hrs by Hoechst 33258 staining-based fluorescence microscopic analysis	Design, synthesis, and biological evaluation of novel combretastatin A-4 thio derivatives as microtubule targeting agents. — Eur J Med Chem
CHEMBL5056161	Functional	Inhibition of tubulin polymerization (unknown origin) assessed as fluorescence intensity measured for 90 mins by DAPI based microplate reader	Design, synthesis and biological evaluation of indole-based [1,2,4]triazolo[4,3-a] pyridine derivatives as novel microtubule polymerization inhibitors. — Eur J Med Chem

Cellosaurus cell lines

3 cell lines: 3 induced pluripotent stem cell

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

Cellosaurus	Name	Category	Sex
CVCL_B3NQ	CHOPi005-A	Induced pluripotent stem cell	Male
CVCL_B3NR	CHOPi006-A	Induced pluripotent stem cell	Male
CVCL_B3NS	CHOPi007-A	Induced pluripotent stem cell	Female

Clinical trials (associated diseases)

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

Trial	Phase	Status	Title
NCT00154830	PHASE4	COMPLETED	Alterations of Functional Activities and Leg Stiffness After Hamstring Lengthening in Cerebral Palsy Children
NCT00432055	PHASE4	COMPLETED	Effects of Botulinum Toxin Type A in Adults With Cerebral Palsy
NCT00549471	PHASE4	TERMINATED	Improvement After Botulinum Toxin Injections to the Arms in Children With Cerebral Palsy
NCT00752934	PHASE4	TERMINATED	Does Oral Baclofen Improve Care and Comfort in Spastic Children in Nursing Homes?
NCT00964639	PHASE4	COMPLETED	Postoperative Pain in Children With Cerebral Palsy After Pelvic and Femoral Osteotomies
NCT01386255	PHASE4	WITHDRAWN	Placebo Controlled Study of Baclofen for GERD in Children With Cerebral Palsy
NCT02546999	PHASE4	COMPLETED	Does Botulinum Toxin A Make Walking Easier in Children With Cerebral Palsy?
NCT02633241	PHASE4	COMPLETED	A Pilot Study of Dexmedetomidine-Propofol in Children Undergoing Magnetic Resonance Imaging
NCT03117322	PHASE4	COMPLETED	Synbiotic, Prebiotics and Probiotics in Children With Cerebral Palsy and Constipation
NCT03648658	PHASE4	UNKNOWN	Paracetamol Study in Patients With Low Muscle Mass
NCT04074265	PHASE4	COMPLETED	Peri-operative Use of a Pain Injection in Pediatric Patients With Cerebral Palsy
NCT04273737	PHASE4	TERMINATED	Amantadine in Treating Cognitive & Motor Impairments in Adolescents and Adults With Cerebral Palsy
NCT04523935	PHASE4	COMPLETED	Excessive Crying in Children With Cerebral Palsy and Communication Deficits
NCT05887765	PHASE4	COMPLETED	Effect of Systematic Dexamethasone on the Duration of Popliteal Nerve Block for Anesthesia After Pediatric Ankle Surgery
NCT06176430	PHASE4	UNKNOWN	Comparison of Twice Weekly Versus Daily Iron Therapy in Treating Anemia in Children With Cerebral Palsy
NCT06189781	PHASE4	RECRUITING	Pain Injection Versus Epidural Anesthesia for Hip Surgery in Pediatric Patients With Cerebral Palsy
NCT00014989	PHASE3	COMPLETED	Beneficial Effects of Antenatal Magnesium Sulfate (BEAM Trial)
NCT00065949	PHASE3	UNKNOWN	Magnesium Sulfate to Prevent Brain Injury in Premature Infants
NCT00367068	PHASE3	COMPLETED	Dutch National ITB Study in Children With Cerebral Palsy
NCT00491894	PHASE3	COMPLETED	Safety and Efficacy Study of Oral Glycopyrrolate Liquid for the Treatment of Pathologic (Chronic Moderate to Severe) Drooling in Pediatric Patients 3 to 18 Years of Age With Cerebral Palsy or Other Neurologic Conditions
NCT00632528	PHASE3	COMPLETED	MEOPA to Improve Physical Therapy Results After Multilevel Surgery
NCT00822029	PHASE3	TERMINATED	Use of Oral Bisphosphonates in the Treatment of Osteoporosis of Non-walking Children With Cerebral Palsy
NCT00922077	PHASE3	COMPLETED	Individualized Neurodevelopmental Treatment
NCT01249417	PHASE3	COMPLETED	Dysport® Pediatric Lower Limb Spasticity Study
NCT01251380	PHASE3	COMPLETED	Dysport® Pediatric Lower Limb Spasticity Follow-on Study
NCT01437644	PHASE3	COMPLETED	The Post-Operative Pain in Cerebral Palsy (POPPIES) Trial
NCT01492608	PHASE3	COMPLETED	Magnesium Sulphate for Preterm Birth (MASP Study)
NCT01603602	PHASE3	COMPLETED	BOTOX® Treatment in Pediatric Upper Limb Spasticity
NCT01603615	PHASE3	COMPLETED	BOTOX® Open-Label Treatment in Pediatric Upper Limb Spasticity
NCT01603628	PHASE3	COMPLETED	BOTOX® Treatment in Pediatric Lower Limb Spasticity
NCT01603641	PHASE3	COMPLETED	BOTOX® Open-Label Treatment in Pediatric Lower Limb Spasticity
NCT01633736	PHASE3	UNKNOWN	Targeted Hip Strength Training in Children With Cerebral Palsy (CP)
NCT01898520	PHASE3	COMPLETED	A Safety, Efficacy and Tolerability Study of Sativex for the Treatment of Spasticity in Children Aged 8 to 18 Years
NCT01929434	PHASE3	COMPLETED	Efficacy of Stem Cell Transplantation Compared to Rehabilitation Treatment of Patients With Cerebral Paralysis
NCT02002884	PHASE3	COMPLETED	Dose-response Study of Efficacy and Safety of Botulinum Toxin Type A to Treat Spasticity of the Arm(s) or of Arm(s) and Leg(s) in Cerebral Palsy
NCT02270736	PHASE3	COMPLETED	Clinical Study to Investigate the Efficacy and Safety of NT 201 Compared to Placebo in the Treatment of Chronic Troublesome Drooling Associated With Neurological Disorders and/or Intellectual Disability
NCT02839785	PHASE3	TERMINATED	Analgesia and Physiotherapy in Children With Cerebral Palsy (ANTALKINECP)
NCT03110341	PHASE3	UNKNOWN	Effect of Erythropoietin in Premature Infants on White Matter Lesions and Neurodevelopmental Outcome
NCT03302871	PHASE3	COMPLETED	Integrated Management Enhances Functional Gains in Children With Cerebral Palsy Treated by BoNT-A
NCT03306212	PHASE3	COMPLETED	Efficacy of Intermittent Serial Casting on Spastic Wrist Flexion Deformity