Hacettepe Childrens Hospital

Ankara 06230

0 studies enrolling now · 3 studies all time

What they study most

Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Muscular Diseases, Muscular Disorders, Atrophic, Muscular Dystrophies, Muscular Dystrophy, Duchenne, Musculoskeletal Diseases, Nervous System Diseases, Neuromuscular Diseases, Spinal Muscular Atrophy

Studies at this site

• A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy — Stopped early
• An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy — Stopped early
• Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) — Completed