Sandhoff Disease
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2026-03-06Clinical Trial Update: Translational Potential of ex Vivo Gene Therapy in GM2 Gangliosidosis
A translational update on ex vivo gene therapy approaches for GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases) with status not yet recru…
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CLINICAL_TRIALS research · 2026-03-04Clinical Trial Update: A Natural History Study of the Gangliosidoses
This clinical trial update describes an ongoing natural history study of gangliosidoses. It includes Tay-Sachs disease, Sandhoff disease, GM…
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CLINICAL_TRIALS research · 2025-09-08Clinical Trial Update: UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells [Phase 1]
Phase 1 trial evaluating intrathecal umbilical cord blood-derived oligodendrocyte-like cells for several inherited metabolic brain diseases;…