Disease · MONDO_0010679

Duchenne muscular dystrophy

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. Duchenne muscular dystrophy. Diseases, record MONDO_0010679. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0010679

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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: Duchenne muscular dystrophy; Open Targets
Open Targets ID: MONDO_0010679; Open Targets
Other names: DMD, Duchenne muscular dystrophy, X-linked recessive, severe dystrophinopathy, Duchenne type; Open Targets
Disease Ontology: DOID:11723; Disease Ontology
MeSH: D020388; MeSH
NCI Thesaurus: C75482; NCI Thesaurus
Orphanet: ORPHA:98896; Orphanet
Highest stage of its drugs: Approval, for 9 of its 62 drugs, from ClinicalTrials.gov, DailyMed, EMA Human Drugs, FDA, PMDA and TTD; Open Targets

Drugs62

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
ATALUREN	CHEMBL256997	Small molecule	Approval	EMA Human Drugs, at withdrawal	14	13	13	Drugs and targets
ETEPLIRSEN	CHEMBL2108278	Oligonucleotide	Approval	FDA	13	11	11	Drugs and targets
VILTOLARSEN	CHEMBL4298062	Oligonucleotide	Approval	ClinicalTrials.gov, FDA and PMDA	12	8	8	Drugs and targets
VAMOROLONE	CHEMBL2348780	Small molecule	Approval	EMA Human Drugs, FDA and TTD	11	8	8	Drugs and targets
GOLODIRSEN	CHEMBL4297762	Oligonucleotide	Approval	FDA	8	6	6	Drugs and targets
DEFLAZACORT	CHEMBL1201891	Small molecule	Approval	ClinicalTrials.gov, DailyMed and FDA	8	5	5	Drugs and targets
CASIMERSEN	CHEMBL4297566	Oligonucleotide	Approval	FDA	7	5	5	Drugs and targets
GIVINOSTAT	CHEMBL1213492	Small molecule	Approval	EMA Human Drugs	6	4	4	Drugs and targets
GIVINOSTAT HYDROCHLORIDE	CHEMBL6068400	Small molecule	Approval	FDA	3	1	1	Drugs and targets
DRISAPERSEN	CHEMBL2108735	Oligonucleotide	Phase 3	ClinicalTrials.gov	9	9	9	Drugs and targets

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Targets184

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
DMD	Dystrophin	8	Dystrophin exogenous gene; Dystrophin pre-mRNA positive modulator	Approval	https://conductscience.org/library/genes/1756	Drugs and targets
NR3C1	Glucocorticoid receptor	4	Glucocorticoid receptor agonist	Approval	https://conductscience.org/library/genes/2908	Drugs and targets
NR3C2	Mineralocorticoid receptor	3	Mineralocorticoid receptor antagonist	Approval	https://conductscience.org/library/genes/4306	Drugs and targets
PDE5A	3',5'-cyclic phosphodiesterase	3	3',5'-cyclic phosphodiesterase inhibitor; Phosphodiesterase 5A inhibitor	Phase 3	https://conductscience.org/library/genes/8654	Drugs and targets
HDAC1	Histone deacetylase	2	Histone deacetylase inhibitor	Approval	https://conductscience.org/library/genes/3065	Drugs and targets
HDAC10	Histone deacetylase	2	Histone deacetylase inhibitor	Approval	https://conductscience.org/library/genes/83933	Drugs and targets
HDAC11	Histone deacetylase	2	Histone deacetylase inhibitor	Approval	https://conductscience.org/library/genes/79885	Drugs and targets
HDAC2	Histone deacetylase	2	Histone deacetylase inhibitor	Approval	https://conductscience.org/library/genes/3066	Drugs and targets
HDAC3	Histone deacetylase	2	Histone deacetylase inhibitor	Approval	https://conductscience.org/library/genes/8841	Drugs and targets
HDAC4	Histone deacetylase	2	Histone deacetylase inhibitor	Approval	https://conductscience.org/library/genes/9759	Drugs and targets

1 to 10 of 184

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Trials145

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. 4 more are not shown, since the checks do not confirm them.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT03179631	Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy	Phase 3	360	2026-03-10	Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Disease, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn	ATALUREN	Its record names the disease	ClinicalTrials.gov
NCT01865084	A Study of Tadalafil for Duchenne Muscular Dystrophy	Phase 3	331	2017-03-01	Muscular Dystrophy, Duchenne	TADALAFIL	Its record names the disease	ClinicalTrials.gov
NCT01826487	Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)	Phase 3	230	2020-08-04	Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn	ATALUREN	Its record names the disease	ClinicalTrials.gov
NCT02090959	An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy	Phase 3	219	2020-08-11	Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn	ATALUREN	Its record names the disease	ClinicalTrials.gov
NCT01603407	Finding the Optimum Regimen for Duchenne Muscular Dystrophy	Phase 3	196	2022-08-12	Duchenne Muscular Dystrophy	DEFLAZACORT, PREDNISONE	Its record names the disease	ClinicalTrials.gov
NCT01254019	A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy	Phase 3	186	2019-01-28	Muscular Dystrophies	DRISAPERSEN	Its record names the disease	ClinicalTrials.gov
NCT02851797	Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy	Phase 3	179	2023-02-02	Duchenne Muscular Dystrophy	GIVINOSTAT	Its record names the disease	ClinicalTrials.gov
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
NCT03532542	An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy	Phase 3	171	2024-09-19	Duchenne Muscular Dystrophy	CASIMERSEN, GOLODIRSEN	Its record names the disease	ClinicalTrials.gov

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Comparisons in its trials48

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
Ataluren vs Placebo: DB Period: Number of Participants With Treatment-emergent Adverse Events (TEAEs)	157 of 184	149 of 176	360	NCT03179631	Trial comparisons
Ataluren/Ataluren vs Placebo/Ataluren: Overall Treatment Period: Number of Participants With TEAEs	170 of 184	110 of 172	356	NCT03179631	Trial comparisons
Ataluren vs Placebo: serious adverse events	4 of 115	4 of 115	230	NCT01826487	Trial comparisons
GSK2402968 6mg/kg/Week vs Placebo: Number of Participants With Abnormal-clinically Significant Electrocardiogram (ECG) Findings at Any Visit Post-Baseline	5 of 125	0 of 61	186	NCT01254019	Trial comparisons
GSK2402968 6mg/kg/Week vs Placebo: deaths from any cause	0 of 125	0 of 61	186	NCT01254019	Trial comparisons
GSK2402968 6mg/kg/Week vs Placebo: serious adverse events	13 of 125	5 of 61	186	NCT01254019	Trial comparisons
Givinostat vs Placebo: deaths from any cause	0 of 118	0 of 61	179	NCT02851797	Trial comparisons
Givinostat vs Placebo: serious adverse events	8 of 118	2 of 61	179	NCT02851797	Trial comparisons
Golodirsen vs Casimersen: Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs)	12 of 74	22 of 97	171	NCT03532542	Trial comparisons
Golodirsen vs Casimersen: deaths from any cause	1 of 74	0 of 97	171	NCT03532542	Trial comparisons

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Papers from its trials83

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
36036925	RESULT	1	PubMed
32956407	RESULT	3	PubMed
31001554	RESULT	1	PubMed
29123500	RESULT	1	PubMed
27566866	RESULT	1	PubMed
26770204	RESULT	1	PubMed
25907158	RESULT	1	PubMed
21435876	RESULT	1	PubMed
21428760	RESULT	1	PubMed
21179598	RESULT	1	PubMed

1 to 10 of 83

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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_0010679

GET https://conductscience.org/api/diseases/v1/MONDO_0010679/drugs?page=2

API reference For AI assistants