Rare Disease Research, LLC

Hillsborough, North Carolina 27278

2 studies enrolling now · 3 studies all time

What they study most

Allan-Herndon-Dudley Syndrome, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DM1, DMD, Duchenne Muscular Dystrophy (DMD), Genetic Disease, Inborn, Genetic Disease, X-Linked, Monocarboxylate Transporter 8 Deficiency, Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Muscular Dystrophy (DMD), Muscular Dystrophy in Children

Studies at this site

• Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1 — Recruiting now
• Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO) — Recruiting now
• Withdrawal of Tiratricol Treatment in Males With Monocarboxylate Transporter 8 Deficiency (MCT8 Deficiency) — Completed