Disease · MONDO_0010311

Becker muscular dystrophy

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. Becker muscular dystrophy. Diseases, record MONDO_0010311. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0010311

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Disease

Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.

Name: Becker muscular dystrophy; Open Targets
Open Targets ID: MONDO_0010311; Open Targets
Other names: BMD, Becker dystrophinopathy, Becker muscular dystrophy, X-linked recessive, Becker's muscular dystrophy, Benign pseudohypertrophic muscular dystrophy, pseudohypertrophic progressive duchenne and becker types muscular dystrophy; Open Targets
Disease Ontology: DOID:9883; Disease Ontology
MeSH: C570377; MeSH
NCI Thesaurus: C84587; NCI Thesaurus
Orphanet: ORPHA:98895; Orphanet
Highest stage of its drugs: Approval, for 1 of its 15 drugs, from FDA; Open Targets

Drugs15

The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.

Drug	ChEMBL ID	Type	Open Targets' stage	Stage reported by	Reports	Of them trials	Trials shown here	Source
DELANDISTROGENE MOXEPARVOVEC	CHEMBL4650241	Gene	Approval	FDA	1	0	0	Drugs and targets
ATALUREN	CHEMBL256997	Small molecule	Phase 3	ClinicalTrials.gov	4	4	4	Drugs and targets
ENALAPRIL	CHEMBL578	Small molecule	Phase 3	ClinicalTrials.gov	1	1	1	Drugs and targets
SEVASEMTEN	CHEMBL5314486	Small molecule	Phase 2	ClinicalTrials.gov	4	4	4	Drugs and targets
(+)-EPICATECHIN	CHEMBL129482	Small molecule	Phase 2	ClinicalTrials.gov	2	2	2	Drugs and targets
(-)-EPICATECHIN	CHEMBL583912	Small molecule	Phase 2	ClinicalTrials.gov	2	2	2	Drugs and targets
GIVINOSTAT	CHEMBL1213492	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
L-CITRULLINE	CHEMBL444814	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
METFORMIN	CHEMBL1431	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
PEMIROLAST	CHEMBL1201198	Small molecule	Phase 2	ClinicalTrials.gov	1	1	0	Drugs and targets

1 to 10 of 15

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Targets146

The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.

Target	Protein	Its drugs that act on it	Mechanisms	Highest stage of those drugs	Gene in Genes	Source
NR3C1	Glucocorticoid receptor	2	Glucocorticoid receptor agonist	Phase 2	https://conductscience.org/library/genes/2908	Drugs and targets
ACE	Angiotensin-converting enzyme	1	Angiotensin-converting enzyme inhibitor	Phase 3	https://conductscience.org/library/genes/1636	Drugs and targets
COXFA4	Mitochondrial complex I (NADH dehydrogenase)	1	Mitochondrial complex I (NADH dehydrogenase) inhibitor	Phase 2	https://conductscience.org/library/genes/4697	Drugs and targets
COXFA4L2	Mitochondrial complex I (NADH dehydrogenase)	1	Mitochondrial complex I (NADH dehydrogenase) inhibitor	Phase 2	https://conductscience.org/library/genes/56901	Drugs and targets
CSF3R	Granulocyte colony-stimulating factor receptor	1	Granulocyte colony stimulating factor receptor agonist	Phase 1	https://conductscience.org/library/genes/1441	Drugs and targets
DMD	Dystrophin	1	Dystrophin exogenous gene	Approval	https://conductscience.org/library/genes/1756	Drugs and targets
FAU	80S Ribosome	1	80S Ribosome modulator	Phase 3	https://conductscience.org/library/genes/2197	Drugs and targets
GPD2	Glycerol-3-phosphate dehydrogenase, mitochondrial	1	Mitochondrial glycerol-3-phosphate dehydrogenase inhibitor	Phase 2	https://conductscience.org/library/genes/2820	Drugs and targets
HDAC1	Histone deacetylase	1	Histone deacetylase inhibitor	Phase 2	https://conductscience.org/library/genes/3065	Drugs and targets
HDAC10	Histone deacetylase	1	Histone deacetylase inhibitor	Phase 2	https://conductscience.org/library/genes/83933	Drugs and targets

1 to 10 of 146

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Trials18

Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs. 1 more is not shown, since the checks do not confirm it.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT00592553	Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)	Phase 2	174	2020-04-07	Duchenne Muscular Dystrophy, Becker Muscular Dystrophy	ATALUREN	Its record names the disease	ClinicalTrials.gov
NCT00847379	Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)	Phase 2	173	2020-07-15	Duchenne Muscular Dystrophy, Becker Muscular Dystrophy	ATALUREN	Its record names the disease	ClinicalTrials.gov
NCT01557400	Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada	Phase 3	94	2020-11-25	Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Dystrophinopathy	ATALUREN	Its record names the disease	ClinicalTrials.gov
NCT03238235	Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Becker Muscular Dystrophy	Phase 2	51	2024-11-18	Becker Muscular Dystrophy	GIVINOSTAT	Its record names the disease	ClinicalTrials.gov
NCT04054375	Weekly Steroids in Muscular Dystrophy	Phase 2	20	2021-07-08	Limb-girdle Muscular Dystrophy, Becker Muscular Dystrophy	PREDNISONE	Its record names the disease	ClinicalTrials.gov
NCT05160415	A Study of EDG-5506 in Adult Males With Becker Muscular Dystrophy	Phase 1	12	2025-06-24	Becker Muscular Dystrophy	SEVASEMTEN	Its record names the disease	ClinicalTrials.gov
NCT01856868	Use of (-)-Epicatechin in the Treatment of Becker Muscular Dystrophy (Pilot Study)	Phase 1/2	7	2021-12-22	Becker Muscular Dystrophy	(+)-EPICATECHIN, (-)-EPICATECHIN	Its record names the disease	ClinicalTrials.gov
NCT01009294	Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)	Phase 2	6	2020-07-29	Duchenne Muscular Dystrophy, Becker Muscular Dystrophy	ATALUREN	Its record names the disease	ClinicalTrials.gov
NCT03236662	(-)- Epicatechin Becker Muscular Dystrophy	Phase 2	2	2021-07-26	Becker Muscular Dystrophy	(+)-EPICATECHIN, (-)-EPICATECHIN	Its record names the disease	ClinicalTrials.gov
NCT06066580	Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy	Phase 2	260		Becker Muscular Dystrophy	SEVASEMTEN	Its record names the disease	ClinicalTrials.gov

1 to 10 of 18

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Comparisons in its trials5

Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.

Comparison	Group 1, with the outcome	Group 2, with the outcome	Participants	Trial	Source
Givinostat vs Placebo: Percentage of Patients Who Fell During the 6MWT	1 of 34	1 of 17	51	NCT03238235	Trial comparisons
Givinostat vs Placebo: Count of Participant Who Lose Ambulation During the Study (From Baseline to Month 12)	0 of 34	0 of 17	51	NCT03238235	Trial comparisons
Givinostat vs Placebo: deaths from any cause	0 of 34	0 of 17	51	NCT03238235	Trial comparisons
Givinostat vs Placebo: Percentage of Patients With < 10% Worsening in 6MWT After 12 Months of Treatment.	7 of 34	1 of 17	51	NCT03238235	Trial comparisons
Givinostat vs Placebo: serious adverse events	0 of 34	0 of 17	51	NCT03238235	Trial comparisons

Papers from its trials9

The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.

PMID	Listed as	Trials listing it	Source
31001554	RESULT	1	PubMed
29123500	RESULT	1	PubMed
26770204	RESULT	1	PubMed
40452637	DERIVED	1	PubMed
37236246	DERIVED	1	PubMed
34791888	DERIVED	1	PubMed
34693725	DERIVED	1	PubMed
32851872	DERIVED	1	PubMed
27926769	DERIVED	1	PubMed

Data dates

The date of each source this release read.

Open Targets: 2026-09-18; Open Targets
Drug links and their checks, from Drugs and targets: 2026-09-18; Drugs and targets
ClinicalTrials.gov, from Trials: 2026-11-12; Trials
Comparisons, from Trial comparisons: 2026-03-23; Trial comparisons
PubMed, for the checks: 2026-11-06; PubMed
MeSH, for the checks: 2026-11-10; MeSH

Sources 6

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ChEMBL, through Open TargetsCC BY-SA 3.0
ClinicalTrials.govNo license stated
NLM's MeSHNLM's terms
Open TargetsCC0 1.0
PubMedNLM's terms

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
ClinicalTrials.govnone stated
NLM's MeSHNLM's
Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
PubMedNLM's terms

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this disease from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/diseases/v1/MONDO_0010311

GET https://conductscience.org/api/diseases/v1/MONDO_0010311/targets?page=2

API reference For AI assistants