Genethon

A Clinical Study to Assess Safety, Efficacy and Pharmacodynamic Profile of GNT0004 in Duchenne Muscular Dystrophy

Role: lead

Multicenter Epidemiological Study for the Evaluation of the Seroprevalence of Total and Neutralizing Antibodies Against Adenoassociated Virus Serotypes Used in Gene Therapy in Patients With Neuromuscular Diseases of Genetic Origin.

Role: lead

Gene Therapy for X-linked Chronic Granulomatous Disease (X-CGD)

Role: lead

Gene Therapy for X-linked Chronic Granulomatous Disease

Role: lead

Natural History Study of Patients With Limb-Girdle Muscular Dystrophy 2I

Role: lead

Natural History of Duchenne Muscular Dystrophy

Role: lead

Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease

Role: collaborator

Efficacy and Safety of GNT0003 Following Imlifidase Pre-treatment in Severe Crigler-Najjar Syndrome

Role: lead

Efficacy Study of Daily Administration of VPA in Patients Affected by Wolfram Syndrome

Role: collaborator

Gene Therapy for Severe Crigler Najjar Syndrome

Role: lead

Gene Therapy for Wiskott-Aldrich Syndrome (WAS)

Role: lead

Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome

Role: lead

Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM)

Role: collaborator

Gene Therapy for WAS

Role: lead

Immunity Against AAV in Crigler Najjar Patient

Role: lead

Research of Biomarkers in Duchenne Muscular Dystrophy Patients

Role: lead

Observational Study of Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping

Role: lead

Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C

Role: lead