N-Acetyl-L-Leucine for GM2 Gangliosdisosis (Tay-Sachs and Sandhoff 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 Gene Therapy for Tay-Sachs Disease Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease) 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