7 studies found for: Open Studies | "Tay-Sachs Disease"
Gene Therapy for Tay-Sachs Disease
Biomarker for GM1/GM2 - Gangliosidoses
A Natural History Study of the Gangliosidoses
Synergistic Enteral Regimen for Treatment of the Gangliosidoses
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
HSCT for High Risk Inherited Inborn Errors
Efficacy Study of an Online Educational Module Before Carrier Genetic Screening in Persons of Ashkenazi Jewish Descent.
† Indicates status has not been verified in more than two years