Clinical Trials

Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1

Sleep Breathing Disorders, a Main Trigger for Cardiac ARythmias in Type I Myotonic Dystrophy ?

Study of Ranolazine in Myotonia Congenita, Paramyotonia Congenita and Myotonic Dystrophy Type 1

Symptoms and Outcome Measures for Upper- Limb Function in Myotonic Dystrophy Type 1

Technology Assisted Rehabilitation for Upper Limb Function in Myotonic Dystrophy Type 1

A Registered Observational Cohort Study of Myotonic Dystrophy Type 1

A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I

Global Open-Label Extension Study of Del-desiran for the Treatment of DM1

Calcium Channel Blocker in Myotonic Dystrophy Type 1

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

Wheelchair Skills Training for People with ARSACS and DM1

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

A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 50 Years of Age With Non-congenital Myotonic Dystrophy Type 1

Cerebrospinal Fluid Biomarkers of Myotonic Dystrophy

DMCRN-02-001: Assessing Pediatric Endpoints in DM1

Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology

Music Intervention for Brain-Heart Disease in Myotonic Dystrophy Type 1 (DM1)

Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry

Pelvic Floor Muscle Training for Women with Myotonic Dystrophy

Personalized Training for People With Rare Neuromuscular Disorders