Search by Medical Condition
A B C D E F G H I J K L M N O P Q R S T U V W X Y ZClinical Trials for Gangliosidoses
- Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
- GM1 Prophylaxis for WBRT Related Cognitive Dysfunction
- A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human <=-Galactosidase in Type II GM1 Gangliosidosis
- Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD)
- N-Acetyl-L-Leucine for GM2 Gangliosdisosis (Tay-Sachs and Sandhoff Disease)
- MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
- Synergistic Enteral Regimen for Treatment of the Gangliosidoses
- Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)
- A Phase I Study of Pyrimethamine in Patients With GM2 Gangliosidosis
- Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses
- Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis
- HSCT for High Risk Inherited Inborn Errors
- Stem Cell Transplant for Inborn Errors of Metabolism