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Home › Conditions › Duchenne muscular dystrophy

Duchenne muscular dystrophy

Part of Muscular Dystrophies

4 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for Duchenne muscular dystrophy

57 trials tracked for Duchenne muscular dystrophy: 13 in phase 3 or 4 and 10 with published results. The most-cited published study has 454 citations.

57Trials tracked
13Phase 3 & 4
0Recruiting
10With published results
Phase distribution
Phase 4 2 Phase 3 11 Phase 2 34 Phase 1 4 Other / NA 6
  1. Phase 3 Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Completed · 454 cited
  2. Phase 3 A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) Completed · 149 cited
  3. Phase 3 Finding the Optimum Regimen for Duchenne Muscular Dystrophy Completed · 123 cited
  4. Phase 3 Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy Completed · 115 cited
  5. Phase 3 Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy Completed · 64 cited
  6. Phase 3 Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada Completed · 62 cited
Show 44 more trials
  1. Phase 4 Stacking Exercises Aid the Decline in FVC and Sick Time Completed · 48 cited
  2. Phase 3 Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy Completed · 30 cited
  3. Phase 3 Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy Completed · 23 cited
  4. Phase 3 Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy Completed
  5. Phase 4 Flu Vaccine Study in Neuromuscular Patients 2011 Completed
  6. Phase 3 Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53) Completed
  7. Phase 3 Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD) Completed
  8. Phase 2 Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy Completed
  9. Phase 2 A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren Completed
  10. Phase 2 A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) Completed
  11. Phase 2 Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy Completed
  12. Phase 2 Halt cardiomyOPathy progrEssion in Duchenne (HOPE-OLE) Completed
  13. Phase 2 Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) Completed
  14. Phase 2 A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Completed
  15. Phase 2 Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy Completed
  16. Phase 2 AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications. Completed
  17. Phase 2 Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory Adolescents Completed
  18. Phase 2 Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy Completed
  19. Phase 2 Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) Completed
  20. Phase 2 Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy Completed
  21. Phase 2 A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD) Completed
  22. Phase 2 Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD) Completed
  23. Phase 2 Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53) Completed
  24. Phase 2 A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications. Completed
  25. Phase 2 HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne) Completed
  26. Phase 2 Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping Completed
  27. Phase 2 Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients Completed
  28. Phase 2 A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) Completed
  29. Phase 2 Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy Completed
  30. Phase 2 Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy Completed
  31. Phase 2 A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy Completed
  32. Phase 2 Study of DS-5141b in Patients With Duchenne Muscular Dystrophy Completed
  33. Phase 2 A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD) Completed
  34. Phase 2 An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) Completed
  35. Phase 2 A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) Completed
  36. Phase 2 Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy Completed
  37. Phase 2 Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients Completed
  38. Phase 2 Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) Completed
  39. Phase 2 Phase I/II Study of SRP-4053 in DMD Patients Completed
  40. Phase 2 A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy Completed
  41. Phase 2 Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) Completed
  42. Phase 1 Safety Study of Transvenous Limb Perfusion in Human Muscular Dystrophy Completed
  43. Phase 1 A Study of TAS-205 for Duchenne Muscular Dystrophy Completed
  44. Phase 1 Rimeporide in Patients With Duchenne Muscular Dystrophy Completed

Showing the 50 most-cited and recently-updated of 57 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
Transition programs for DMD vary widely with significant gaps in policy-driven implementation and neurodiversity support
Primary Care & Family Medicine Meta-analysis
Transition programs for DMD vary widely with significant gaps in policy-driven implementation and neurodiversity support Health systems for young people with Duchenne muscular dystrophy need better support and clear rules
This systematic review with narrative synthesis examines transition-related health systems for youth and adults living with Duchenne muscula…
A review of 42 studies shows care plans for teens with Duchenne muscular dystrophy vary widely. Most programs are temporary projects instead…
Frontiers May 29, 2026
Tamoxifen adjunct to corticosteroids shows no motor benefit in Duchenne muscular dystrophy extension study
Pediatrics RCT
Tamoxifen adjunct to corticosteroids shows no motor benefit in Duchenne muscular dystrophy extension study Does adding tamoxifen to standard treatment help boys with Duchenne muscular dystrophy?
A 48-week open-label extension of a phase 3 trial in 66 boys with DMD found no significant difference in motor function change between early…
Adding tamoxifen to standard steroid treatment did not improve motor function or slow disease progression in boys with Duchenne muscular dys…
Apr 3, 2026
Phase 3 RCT: Ataluren shows favorable trend for slowing functional decline in nonsense mutation DMD
Pediatrics RCT
Phase 3 RCT: Ataluren shows favorable trend for slowing functional decline in nonsense mutation DMD Can a drug help boys with Duchenne muscular dystrophy walk longer?
A phase 3 RCT of 359 people with nonsense mutation Duchenne muscular dystrophy found that 72 weeks of ataluren treatment was associated with…
Boys with a specific genetic form of Duchenne muscular dystrophy who took ataluren saw their walking ability decline less over 72 weeks than…
Apr 3, 2026
Phase 2 trial of oral ifetroban in DMD cardiomyopathy reports no safety results yet
Cardiology Phase II
Phase 2 trial of oral ifetroban in DMD cardiomyopathy reports no safety results yet Can a new pill help protect the hearts of boys with Duchenne muscular dystrophy?
A phase 2 randomized, double-blind, placebo-controlled trial evaluated low- and high-dose oral ifetroban in 46 males with DMD-associated car…
A new pill called ifetroban is being tested to safely treat heart weakness in boys with Duchenne muscular dystrophy, addressing a leading ca…
CT.gov Apr 2, 2026

Research across Muscular Dystrophies

Related studies from across the Muscular Dystrophies family.

Russian FSHD registry shows moderate inverse correlation between D4Z4 repeats and clinical severity
Genetics & Precision Medicine Cohort
Russian FSHD registry shows moderate inverse correlation between D4Z4 repeats and clinical severity What does FSHD look like in Russia? A registry study maps the disease
An observational cohort study of 470 patients in the Russian FSHD Patient Registry found genetic confirmation in 76% of participants.
Nearly half of Russian FSHD patients first notice shoulder blade weakness, while others start with facial symptoms or a classic childhood fo…
medRxiv Apr 8, 2026