5 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
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
Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)
Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases
† Indicates status has not been verified in more than two years