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 · 2025-10-16Long-Term Follow-Up of A Two-Stage Dose-Escalation Study to Evaluate the Safety and Efficacy of Bilateral Intraparenchymal Thalamic and Intracisternal/Intrathecal Admin of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease
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CLINICAL_TRIALS study record update · 2023-04-12Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
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CLINICAL_TRIALS study record update · 2019-07-11Treatment of High Risk, Inherited Lysosomal And Peroxisomal Disorders by Reduced Intensity Hematopoietic Stem Cell Transplantation
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CLINICAL_TRIALS study record update · 2017-08-17Assessing the Outcomes of Web-based Pre-test Educational Module for Carrier Genetic Screening in Individuals of Ashkenazi Jewish Descent
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CLINICAL_TRIALS study record update · 2014-12-04Gene Therapy for Tay-Sachs Disease (Phase 1: Natural History Data Gather)
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CLINICAL_TRIALS study record update · 2011-06-10Treatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Bone Marrow Transplantation