Gene Therapy for Tay-Sachs Disease

Tay Sachs disease

GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO) A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease Registry Gangliosidoses Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2 Efficacy Study of an Online Educational Module Before Carrier Genetic Screening in Persons of Ashkenazi Jewish Descent. Diagnostic and Screening Study of Genetic Disorders Longitudinal Study of Neurodegenerative Disorders A Natural History Study of the Gangliosidoses N-Acetyl-L-Leucine for GM2 Gangliosdisosis (Tay-Sachs and Sandhoff Disease) Synergistic Enteral Regimen for Treatment of the Gangliosidoses Gene Therapy for Tay-Sachs Disease Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)