Rare Disease Research, LLC
Rare Disease Research, LLC
Hillsborough, North Carolina, United States2 results ยท Page 1 of 1
Clinical trials
Swipe the table sideways for more columns.
| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, Myotonia, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic dystrophy type 1, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Recruiting | Phase 3 | 2026-10-05 | NCT07486934 | |
| Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne and Becker Muscular Dystrophy, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Recruiting | Phase 3 | 2026-05-27 | NCT07608432 |