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Clinical trial condition

Myotonic Dystrophy

Explore clinical trials studying Myotonic Dystrophy. Study availability and eligibility vary by location and protocol.

107 public trials 37 recruiting studies
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Myotonic Dystrophy 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 34 locations
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Recruiting

A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping

NCT07587242

Conditions
Congenital, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DMD, +22 more
Locations
  • AP-HP Hospital Armand-Trousseau Paris, France
  • Universitaetsklinikum Essen Essen, Germany
  • Universitaetsklinikum Heidelberg Heidelberg, Germany
  • Klinikum der Ludwig-Maximilians-Universitaet Muenchen München, Germany
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Recruiting

Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension

NCT06747884

Conditions
Childhood Myotonic Dystrophy, Congenital Myotonic Dystrophy, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +16 more
Locations
  • Virginia Commonwealth University Richmond, Virginia, United States
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • University of Kansas Medical Center Kansas City, Kansas, United States
  • Hospital das Clínicas, University of São Paulo School of Medicine (HCFMUSP) Cerqueira César, São Paulo, Brazil
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Recruiting

Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy

NCT06138743

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, +10 more
Locations
  • Liverpool Hospital Liverpool, New South Wales, Australia
  • Sunshine Coast University Hospital Birtinya, Queensland, Australia
  • Royal Brisbane And Women's Hospital Herston, Queensland, Australia
  • Alfred Health Melbourne, Victoria, Australia
View all 35 locations
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Recruiting

Personalized Training for People With Rare Neuromuscular Disorders

NCT06708468

Conditions
Charcot Marie Tooth Disease (CMT), Charcot-Marie-Tooth Disease, Congenital Abnormalities, +20 more
Locations
  • Haukeland University Hospital Bergen, Norway
  • Helse Møre and Romsdal HF (Molde Hospital) Molde, Norway
  • Namsos Hospital Namsos, Norway
  • Oslo University Hospital Oslo, Norway
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Recruiting

DMCRN-02-001: Assessing Pediatric Endpoints in DM1

NCT05224778

Conditions
CDM, Congenital Myotonic Dystrophy, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +11 more
Locations
  • University of California, Los Angeles Los Angeles, California, United States
  • University of Kansas Medical Center Fairway, Kansas, United States
  • University of Rochester Medical Center Rochester, New York, United States
  • Virginia Commonwealth University Richmond, Virginia, United States
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Recruiting

Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)

NCT03981575

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DM1, Genetic Diseases, Inborn, +11 more
Locations
  • University of California, San Diego La Jolla, California, United States
  • University of California, Los Angeles Los Angeles, California, United States
  • University of Colorado - Denver Denver, Colorado, United States
  • University of Florida Gainesville, Florida, United States
View all 17 locations
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Recruiting

A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)

NCT06185764

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, +15 more
Locations
  • Stanford Neuromuscular Research San Carlos, California, United States
  • University of Florida Clinical Research Center Gainesville, Florida, United States
  • University of Kansas Medical Center Fairway, Kansas, United States
  • Boston Children's Hospital Boston, Massachusetts, United States
View all 26 locations
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