Neurology Rare Disease Center

Flower Mound, Texas 75082

6 studies enrolling now · 8 studies all time

What they study most

Anti-myostatin, Duchenne Muscular Dystrophy, Muscular Atrophy, Spinal, Neuromuscular Manifestations, Pediatric Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), SMA, Spinal Muscular Atrophy, Spinal Muscular Atrophy (SMA), Spinal Muscular Atrophy Type 2, Spinal Muscular Atrophy Type 3

Studies at this site

• A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA) — Recruiting now
• A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy — Recruiting now
• A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE) — Recruiting now
• 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 — Recruiting now
• Two Dose Levels of Privigen in Pediatric CIDP — Recruiting now
• A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy — Recruiting now
• Phase 2 Expanded Access Study of UC-MSC in DMD Patients — Running, not enrolling
• A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD) — Running, not enrolling