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Disease · MONDO_0010679

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.

TrialTitlePhaseParticipantsResults first postedConditionsDrugsLinked bySource
NCT03179631Long-Term Outcomes of Ataluren in Duchenne Muscular DystrophyPhase 33602026-03-10
Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Disease, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
ATALURENIts record names the diseaseClinicalTrials.gov
NCT01865084A Study of Tadalafil for Duchenne Muscular DystrophyPhase 33312017-03-01Muscular Dystrophy, DuchenneTADALAFILIts record names the diseaseClinicalTrials.gov
NCT01826487Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)Phase 32302020-08-04
Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
ATALURENIts record names the diseaseClinicalTrials.gov
NCT02090959An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation DystrophinopathyPhase 32192020-08-11
Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
ATALURENIts record names the diseaseClinicalTrials.gov
NCT01603407Finding the Optimum Regimen for Duchenne Muscular DystrophyPhase 31962022-08-12Duchenne Muscular DystrophyDEFLAZACORT, PREDNISONEIts record names the diseaseClinicalTrials.gov
NCT01254019A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular DystrophyPhase 31862019-01-28Muscular DystrophiesDRISAPERSENIts record names the diseaseClinicalTrials.gov
NCT02851797Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular DystrophyPhase 31792023-02-02Duchenne Muscular DystrophyGIVINOSTATIts record names the diseaseClinicalTrials.gov
NCT00592553Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)Phase 21742020-04-07Duchenne Muscular Dystrophy, Becker Muscular DystrophyATALURENIts record names the diseaseClinicalTrials.gov
NCT00847379Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)Phase 21732020-07-15Duchenne Muscular Dystrophy, Becker Muscular DystrophyATALURENIts record names the diseaseClinicalTrials.gov
NCT03532542An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular DystrophyPhase 31712024-09-19Duchenne Muscular DystrophyCASIMERSEN, GOLODIRSENIts record names the diseaseClinicalTrials.gov
NCT03039686Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular DystrophyPhase 2/31662020-12-21Duchenne Muscular DystrophyTALDEFGROBEP ALFAIts record names the diseaseClinicalTrials.gov
NCT03703882Phase III Study of Edasalonexent in Boys With Duchenne Muscular DystrophyPhase 31312022-06-21Muscular Dystrophy, DuchenneEDASALONEXENTIts record names the diseaseClinicalTrials.gov
NCT02310763A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular DystrophyPhase 21212019-07-23Duchenne Muscular DystrophyDOMAGROZUMABIts record names the diseaseClinicalTrials.gov
NCT03439670A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)Phase 21212022-07-13Duchenne Muscular DystrophyVAMOROLONEIts record names the diseaseClinicalTrials.gov
NCT02255552Study of Eteplirsen in DMD PatientsPhase 31092020-07-01Duchenne Muscular Dystrophy (DMD)ETEPLIRSENIts record names the diseaseClinicalTrials.gov
NCT04371666Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duchenne Muscular Dystrophy (DMD)Phase 3982024-03-12Duchenne Muscular DystrophyPAMREVLUMABIts record names the diseaseClinicalTrials.gov
NCT01557400Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and CanadaPhase 3942020-11-25Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, DystrophinopathyATALURENIts record names the diseaseClinicalTrials.gov
NCT04060199Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)Phase 3772024-12-11Duchenne Muscular DystrophyVILTOLARSENIts record names the diseaseClinicalTrials.gov
NCT04632940Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMDPhase 3732024-08-26Duchenne Muscular DystrophyPAMREVLUMABIts record names the diseaseClinicalTrials.gov
NCT01027884Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)Phase 3652015-10-19Muscular Dystrophy, Duchenne, Ambulatory CareIDEBENONEIts record names the diseaseClinicalTrials.gov

diseases-2026-09-18-0.1.0

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.

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