10 studies found for: "GM2-gangliosidosis, AB variant" OR "Gangliosidoses GM2" OR "Tay-Sachs Disease, AB Variant"
Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis
Biomarker for GM1/GM2 - Gangliosidoses
Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)
A Natural History of Late Onset Tay-Sachs Disease
Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses
A Phase I Study of Pyrimethamine in Patients With GM2 Gangliosidosis
Synergistic Enteral Regimen for Treatment of the Gangliosidoses
A Natural History Study of the Gangliosidoses
Nervous System Degeneration in Glycosphingolipid Storage Disorders
|10||Active, not recruiting||
Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases
† Study has passed its completion date and status has not been verified in more than two years.