Rare Disease Research

Kissimmee, Florida 34746

1 studies enrolling now · 2 studies all time

What they study most

Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Lennox Gastaut Syndrome, Muscular Diseases, Muscular Disorders, Atrophic, Muscular Dystrophies, Muscular Dystrophy, Duchenne, Musculoskeletal Diseases, Nervous System Diseases, Neuromuscular Diseases

Studies at this site

• An Efficacy and Safety Study of Clemizole HCl in Patients With Lennox-Gastaut Syndrome — Recruiting now
• Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping — Available (expanded access)