Records containing: Tay-Sachs Disease
This is the wording recorded or indexed by the source. An agency index term is not necessarily an authorised indication. Co-listed drug names are not linked to this term as a treatment relationship.
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CLINICAL_TRIALS study record update · 2026-03-04A Natural History Study of the Gangliosidoses
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CLINICAL_TRIALS study record update · 2026-02-09Longitudinal Study of Neurodegenerative Disorders
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CLINICAL_TRIALS study record update · 2026-01-28A Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, Safety, and Tolerability of Venglustat in Late-onset GM2 Gangliosidosis (Tay-Sachs Disease and Sandhoff Disease) Together With a Separate Basket for Juvenile/Adolescent Late-onset GM2 Gangliosidosis and Ultra-rare Diseases Within the Same and Similar Glucosylceramide-based Sphingolipid Pathway
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CLINICAL_TRIALS study record update · 2025-09-29Prospective Longitudinal Study of Neurological Disease Trajectory in Children Living With Late-Infantile or Juvenile Onset of GM1 or GM2 Gangliosidosis
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CLINICAL_TRIALS study record update · 2025-09-08Augmentation of Umbilical Cord Blood Transplantation for Inherited Metabolic Diseases With Intrathecal Administration of Human Umbilical Cord Blood-Derived Oligodendrocyte-Like Cells
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CLINICAL_TRIALS study record update · 2025-05-22A Two-Stage, Dose-Escalation and Safety & Efficacy Study of Bilateral Intraparenchymal Thalamic and Intracisternal/Intrathecal Administration of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease
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CLINICAL_TRIALS study record update · 2024-03-12Effects of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease): A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study
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CLINICAL_TRIALS study record update · 2021-11-08Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2
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CLINICAL_TRIALS study record update · 2021-04-14Synergistic Enteral Regimen for Treatment of the Gangliosidoses (Syner-G)
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CLINICAL_TRIALS study record update · 2020-11-12Eight At One Stroke: Attention Gangliosidoses A Registry Study for Patients With Gangliosidoses
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CLINICAL_TRIALS study record update · 2012-02-23Proposed Investigator-Initiated Clinical Trial of Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)