Tay-sachs Disease
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CLINICAL_TRIALS research · 2026-03-30Clinical Trial Update: Caregiving Networks Across Disease Context and the Life Course
The National Human Genome Research Institute sponsors a study examining caregiving networks across disease contexts and life stages, current…
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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…