Intelligence for Neuromuscular & Pediatric Specialists

Duchenne muscular dystrophy clinical trials
recruiting in the United States and worldwide

Up-to-date overview of clinical trials in Duchenne muscular dystrophy (DMD) — AAV microdystrophin gene therapy, exon-skipping antisense oligonucleotides, gene editing, anti-inflammatory and other investigational therapies recruiting patients in the US and worldwide — automatically compiled from ClinicalTrials.gov. Refer eligible patients to participating research centers.

13
Active trials
72
US sites
3
Indications covered
25
Global trials
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13 studies found

Fase 1Duchenne Muscular DystrophyUS 6 sites in the US

A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort

Sarepta Therapeutics, Inc.

📍 Flower Mound, Little Rock, Norfolk, Palo Alto, Sacramento, St Louis👥 83 participants📅 Completion: 2028-02
View details →Refer patient →ClinicalTrials.gov ↗
Fase 2Distrofia Muscular de DuchenneUS 13 sites in the US

AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)

REGENXBIO Inc.

📍 Atlanta, Aurora, Chicago, Cincinnati, Dallas, Gainesville, Iowa City, Little Rock👥 65 participants📅 Completion: 2028-08
View details →Refer patient →ClinicalTrials.gov ↗
Fase 1Duchenne Muscular DystrophyUS 12 sites in the US

A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

Solid Biosciences Inc.

📍 Atlanta, Chicago, Columbus, Little Rock, Los Angeles, Norfolk, Philadelphia, Portland👥 60 participants📅 Completion: 2031-05
View details →Refer patient →ClinicalTrials.gov ↗
Fase 1Dmd Associated Dilated CardiomyopathyUS 3 sites in the US

Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy

Sardocor Corp.

📍 Cincinnati, Columbus, Kansas City👥 12 participants📅 Completion: 2030-10
View details →Refer patient →ClinicalTrials.gov ↗
Fase 1Duchenne Muscular DystrophyUS 10 sites in the US

A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)

Insmed Gene Therapy LLC

📍 Atlanta, Columbus, Davis, Little Rock, Los Angeles, Memphis, Norfolk, Palo Alto👥 12 participants📅 Completion: 2028-03
View details →Refer patient →ClinicalTrials.gov ↗
Fase 3Duchenne Muscular DystrophyUS 1 site in the US

Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)

Dyne Therapeutics

📍 Hillsborough👥 90 participants📅 Completion: 2032-10
View details →Refer patient →ClinicalTrials.gov ↗
Fase 1Duchenne Muscular DystrophyUS 2 sites in the US

Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)

Wave Life Sciences USA, Inc.

📍 Atlanta, Little Rock👥 26 participants📅 Completion: 2027-04
View details →Refer patient →ClinicalTrials.gov ↗
Fase 2Duchenne Muscular DystrophyUS 10 sites in the US

NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)

NS Pharma, Inc.

📍 Atlanta, Aurora, Chicago, Cincinnati, Dallas, Kansas City, Kissimmee, Philadelphia👥 20 participants📅 Completion: 2026-09
View details →Refer patient →ClinicalTrials.gov ↗
Fase 1Duchenne Muscular DystrophyUS 2 sites in the US

PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)

Precision BioSciences, Inc.

📍 Little Rock, St Louis👥 18 participants📅 Completion: 2029-12
View details →Refer patient →ClinicalTrials.gov ↗
Fase 2Duchenne Muscular DystrophyUS 7 sites in the US

Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients

Satellos Bioscience, Inc.

📍 Aurora, Chicago, Columbus, Los Angeles, Seattle, St Louis, Worcester👥 51 participants📅 Completion: 2027-06
View details →Refer patient →ClinicalTrials.gov ↗
Fase 2Duchenne Muscular DystrophyUS 1 site in the US

Vasodilator and Exercise Study for DMD (VASO-REx)

University of Florida

📍 Gainesville👥 50 participants📅 Completion: 2026-11
View details →Refer patient →ClinicalTrials.gov ↗
Fase 1Duchenne Muscular DystrophyUS 1 site in the US

Trial of Cell Based Therapy for DMD

Masonic Cancer Center, University of Minnesota

📍 Minneapolis👥 8 participants📅 Completion: 2027-03
View details →Refer patient →ClinicalTrials.gov ↗
Fase 4Duchenne Muscular DystrophyUS 4 sites in the US

Once Weekly Infant Corticosteroid Trial for DMD

Anne M. Connolly

📍 Chicago, Cincinnati, Columbus, Dallas👥 26 participants📅 Completion: 2026-12
View details →Refer patient →ClinicalTrials.gov ↗

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