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Clinical trial condition

Muscular Dystrophies

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

457 public trials 86 recruiting studies
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Muscular Dystrophies trial results

Recruiting

Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1

NCT07486934

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DM1, Genetic Diseases, Inborn, +18 more
Locations
  • UCSD - Altman Clinical and Translational Research Institute La Jolla, California, United States
  • Rare Disease Research, LLC Atlanta, Georgia, United States
  • IU Health Neuroscience Center Indianapolis, Indiana, United States
  • Roy Blunt NextGen Precision Health Institute Columbia, Missouri, United States
View all 39 locations
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Recruiting

The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2

NCT06523400

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, +9 more
Locations
  • Laboratory for Muscle Diseases and Neuropathies Leuven, Belgium
  • Aarhus University Hospital Aarhus, Denmark
  • Ludug-Maximilians University München, Germany
  • Azienda Ospedaliera Universitaria Policlinico Tor Vergata Rome, Italy
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Recruiting

A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1

NCT06844214

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, +9 more
Locations
  • University of Florida, 2004 Mowry Road - Site Number: 8400005 Gainesville, Florida, United States
  • University of South Florida - Neuromuscular Research, 13330 USF Laurel Drive - Site Number: 8400001 Tampa, Florida, United States
  • Columbia University Medical Center - Neurological Institute, 710 W. 168th, 2nd floor, suite 204 - Site Number : 8400003 New York, United States
  • Hospital Italiano de Buenos Aires, Juan Domingo Peron 4190 - Site Number: 0320001 Buenos Aires, Argentina
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Recruiting

Observatoire Des Patients Atteints de Laminopathies et Emerinopathies (Observatory for PAtients With Laminopathies and Emerinopathies)

NCT03058185

Conditions
Autosomal Emery-Dreifuss Muscular Dystrophy, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Emerinopathies, +11 more
Locations
  • Centre de référence maladies neuromusculaires,CHU d'Angers Angers, France
  • CHU Strasbourg Strasbourg, Bas-Rhin, France
  • CHU Marseille Marseille, Bouches-du-Rhône, France
  • CHU Caen Caen, Calvados, France
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Recruiting

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

NCT04626674

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
  • Stanford University Palo Alto, California, United States
  • University of California, Davis Sacramento, California, United States
  • Washington University in St. Louis St Louis, Missouri, United States
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Recruiting

The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First Three Years in Children With Duchenne Muscular Dystrophy

NCT07092540

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy (DMD), Genetic Diseases, Inborn, +8 more
Locations
  • University of Rochester Rochester, New York, United States
  • Stony Brook Medicine Stony Brook, New York, United States
  • Cincinnati Children's Hospital Medical Center Cincinnati, Ohio, United States
  • Nationwide Children's Hospital Columbus, Ohio, United States
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Recruiting

A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45

NCT07038824

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy (DMD), Genetic Diseases, Inborn, +8 more
Locations
  • University Hospital Gent Ghent, Belgium
  • UZ Leuven Leuven, Belgium
  • Centre Hospitalier Régional de la Citadelle Liège, Belgium
  • IRCCS Ospedale San Raffaele Milan, Italy
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Recruiting

A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44

NCT07037862

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy (DMD), Genetic Diseases, Inborn, +8 more
Locations
  • University Hospital Gent Ghent, Belgium
  • UZ Leuven Leuven, Belgium
  • Centre Hospitalier Régional de la Citadelle Liège, Belgium
  • IRCCS Ospedale San Raffaele Milan, Italy
View all 14 locations
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Recruiting

A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)

NCT07160634

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
  • Neurology Rare Disease Center Flower Mound, Texas, United States
  • Children's Hospital of the King's Daughters Norfolk, Virginia, United States
  • The Children's Hospital of Westmead Sydney, New South Wales, Australia
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