Disease · MONDO_0020121
muscular dystrophy
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. muscular dystrophy. Diseases, record MONDO_0020121. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0020121
On this page
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.
- Other names
Dystrophies, Muscular, Dystrophy, Muscular, HERED PROG MUSC DYSTRPHY, Hereditary progressive muscular dystrophy, Hereditary progressive muscular dystrophy (disorder), Hereditary progressive muscular dystrophy NOS, Hereditary progressive muscular dystrophy NOS (disorder), MD - Muscular dystrophy, Muscular Dystrophies, Muscular dystrophy (disorder), Muscular dystrophy, NOS, Myodystrophica, Myodystrophicas, Myodystrophies, Myodystrophy, PMD - Progressive muscular dystrophy, Progressive muscular dystrophy
- Open Targets
Drugs22
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 |
|---|---|---|---|---|---|---|---|---|
| CASIMERSEN | CHEMBL4297566 | Oligonucleotide | Approval | DailyMed | 1 | 0 | 0 | Drugs and targets |
| DEFLAZACORT | CHEMBL1201891 | Small molecule | Approval | DailyMed | 1 | 0 | 0 | Drugs and targets |
| ETEPLIRSEN | CHEMBL2108278 | Oligonucleotide | Approval | DailyMed | 1 | 0 | 0 | Drugs and targets |
| GOLODIRSEN | CHEMBL4297762 | Oligonucleotide | Approval | DailyMed | 1 | 0 | 0 | Drugs and targets |
| VILTOLARSEN | CHEMBL4298062 | Oligonucleotide | Approval | DailyMed | 1 | 0 | 0 | Drugs and targets |
| SILDENAFIL | CHEMBL192 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| TADALAFIL | CHEMBL779 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| ATALUREN | CHEMBL256997 | Small molecule | Phase 2 | TTD | 1 | 0 | 0 | Drugs and targets |
| DOMAGROZUMAB | CHEMBL3544991 | Antibody | Phase 2 | TTD | 1 | 0 | 0 | Drugs and targets |
| EFMITERMANT ALFA | CHEMBL4297867 | Protein | Phase 2 | TTD | 1 | 0 | 0 | Drugs and targets |
1 to 10 of 22
See all 22 drugsTargets124
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 pre-mRNA | 4 | Dystrophin pre-mRNA positive modulator | Approval | https://conductscience.org/library/genes/1756 | Drugs and targets |
| MSTN | Growth/differentiation factor 8 | 2 | Growth/differentiation factor 8 inhibitor | Phase 2 | https://conductscience.org/library/genes/2660 | Drugs and targets |
| PDE5A | cGMP-specific 3',5'-cyclic phosphodiesterase | 2 | Phosphodiesterase 5A inhibitor | Phase 2 | https://conductscience.org/library/genes/8654 | Drugs and targets |
| ADRM1 | 26S proteasome | 1 | 26S proteasome inhibitor | Phase 1 | https://conductscience.org/library/genes/11047 | Drugs and targets |
| FAU | 80S Ribosome | 1 | 80S Ribosome modulator | Phase 2 | https://conductscience.org/library/genes/2197 | Drugs and targets |
| MAOA | Monoamine oxidase | 1 | Monoamine oxidase inhibitor | Preclinical | https://conductscience.org/library/genes/4128 | Drugs and targets |
| MAOB | Monoamine oxidase | 1 | Monoamine oxidase inhibitor | Preclinical | https://conductscience.org/library/genes/4129 | Drugs and targets |
| MAS1 | Proto-oncogene Mas | 1 | Proto-oncogene Mas agonist | Phase 1 | https://conductscience.org/library/genes/4142 | Drugs and targets |
| NR3C1 | Glucocorticoid receptor | 1 | Glucocorticoid receptor agonist | Approval | https://conductscience.org/library/genes/2908 | Drugs and targets |
| NR3C2 | Mineralocorticoid receptor | 1 | Mineralocorticoid receptor antagonist | Unknown | https://conductscience.org/library/genes/4306 | Drugs and targets |
1 to 10 of 124
See all 124 targetsTrials9
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.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT01521546 | Eplerenone for Subclinical Cardiomyopathy in Duchenne Muscular Dystrophy | Not applicable | 42 | 2015-05-27 | Duchenne Muscular Dystrophy | EPLERENONE | Its record names the disease | ClinicalTrials.gov |
| NCT02239224 | Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Participants With Muscular Dystrophy | Phase 1/2 | 20 | 2021-08-11 | Facioscapulohumeral Muscular Dystrophy (FSHD) | ATYR-1940 | Its record names the disease | ClinicalTrials.gov |
| NCT02531217 | Safety, Tolerability, Pharmacokinetics (PK), and Activity of ATYR1940 in Participants With Muscular Dystrophy - Study Extension | Phase 1/2 | 9 | 2023-08-18 | Facioscapulohumeral Muscular Dystrophy | ATYR-1940 | Its record names the disease | ClinicalTrials.gov |
| NCT00104078 | Study Evaluating MYO-029 in Adult Muscular Dystrophy | Phase 1/2 | 108 | Becker Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy, Limb-Girdle Muscular Dystrophy | STAMULUMAB | Its record names the disease | ClinicalTrials.gov | |
| NCT06290713 | Vasodilator and Exercise Study for DMD (VASO-REx) | Phase 2 | 50 | Duchenne Muscular Dystrophy, Duchenne Disease, Muscular Dystrophy, Muscular Dystrophy in Children, Vasodilation, Exercise, DMD | TADALAFIL | Its record names the disease | ClinicalTrials.gov | |
| NCT04386304 | Safety and Biomarker Response to (+)-Epicatechin in Becker Muscular Dystrophy | Phase 1 | 22 | Becker Muscular Dystrophy | (+)-EPICATECHIN | Its record names the disease | ClinicalTrials.gov | |
| NCT01350154 | Effect of Modulating the nNOS System on Cardiac, Muscular and Cognitive Function in Becker Muscular Dystrophy Patients | Phase 2 | 17 | Becker Muscular Dystrophy | SILDENAFIL | Its record names the disease | ClinicalTrials.gov | |
| NCT02434627 | Sodium Nitrate for Muscular Dystrophy | Phase 1 | 5 | Becker Muscular Dystrophy | SODIUM NITRATE | Its record names the disease | ClinicalTrials.gov | |
| NCT01863004 | Proteasomal Inhibition for Patients With Mis-sense Mutated Dysferlin | Phase 1 | 3 | Dysferlinopathy | BORTEZOMIB | Its record names the disease | ClinicalTrials.gov |
Comparisons in its trials1
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 |
|---|---|---|---|---|
| Eplerenone vs Placebo: serious adverse events | 0 of 20 | 1 of 22 | 42 | NCT01521546 |
Source Trial comparisons
Papers from its trials6
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
Data dates
The date of each source this release read.
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_0020121
GET https://conductscience.org/api/diseases/v1/MONDO_0020121/drugs?page=2