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Clinical trial condition

Muscular Dystrophies

Explore clinical trials studying Muscular Dystrophies. Study availability and eligibility vary by location and protocol.

452 public trials 90 recruiting studies
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Muscular Dystrophies trial results

Active, Not Recruiting

Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

NCT04281485

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • Arkansas Children's Little Rock, Arkansas, United States
  • UCLA Medical Center Los Angeles, California, United States
  • University of Florida Gainesville, Florida, United States
View all 53 locations
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Active, Not Recruiting

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

NCT05693142

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • Children's Hospital of Orange County Orange, California, United States
  • Stanford School of Medicine /Division of Neuromuscular Medicine Palo Alto, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
View all 23 locations
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Active, Not Recruiting

Early Check: Expanded Screening in Newborns

NCT03655223

Conditions
17 Alpha-Hydroxylase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency, +456 more
Locations
  • RTI International Research Triangle Park, North Carolina, United States
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Active, Not Recruiting

A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy

NCT06280209

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • Fondazione Serena ETS - Centro Clinico NeMO Milano Milan, Italy
  • UOC Fase I - Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore Rome, Italy
  • Leids Universitair Medisch Centrum Leiden, Netherlands
  • Hospital Sant Joan de Deu Barcelona, Spain
View Trial Details
Active, Not Recruiting

A Study to Evaluate RO7204239 in Participants With Facioscapulohumeral Muscular Dystrophy

NCT05548556

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Facioscapulohumeral Muscular Dystrophy (FSHD), Genetic Diseases, Inborn, +7 more
Locations
  • University of Irvine Medical Center (UCIMC) Orange, California, United States
  • Regents of the University of Colorado Aurora, Colorado, United States
  • University of Kansas Medical Center Fairway, Kansas, United States
  • Kennedy Krieger Institute Baltimore, Maryland, United States
View Trial Details

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