Gene Therapy :: Nationwide Children's Hospital

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Gene Therapy Clinical Studies

Investigators with the Center for Gene Therapy currently are conducting numerous clinical research studies, described in detail below. Neurosciences at Nationwide Children's offers patients and families a comprehensive approach to the care for children and adolescents with disorders of the brain, spine and nervous system; from initial diagnosis and treatment to rehabilitation and long-term follow-up care.

Learn more about Neurosciences at Nationwide Children's Hospital 

Prosensa Natural History Study (Pro DMD Study)

Phase 3 Study of Ataluren in Patients with Nonsense Mutation Duchenne Muscular Dystrophy

Natural History Study in Mucopolysaccharidosis Type III

Clinical Outcomes Studies in Becker Muscular Dystrophy (BMD) and Sporadic Inclusion Body Myositis (sIBM)

FOR Duchenne Muscular Dystrophy Study (FOR DMD Study)

Nationwide Children's Hospital
700 Children's Drive Columbus, Ohio 43205 614.722.2000