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sandhoff disease

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STUDY
CONDITIONS
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LAST UPDATED

GM1 and GM2 Gangliosidosis PROspective Neurological Disease

Recruiting
  • GM1 Gangliosidosis
  • +2 more
    • Oakland, California
    • +12 more
    Nov 16, 2022

    Adrenoleukodystrophy, Batten Disease, Mucopolysaccharidosis II Trial in Durham (DUOC-01)

    Recruiting
    • Adrenoleukodystrophy
    • +11 more
    • DUOC-01
    • Durham, North Carolina
      Duke University Medical Center
    Oct 24, 2022

    A Natural History Study of the Gangliosidoses

    Recruiting
    • Tay-Sachs Disease
    • +4 more
      • Minneapolis, Minnesota
        University of Minnesota - Pediatric Genetics and Metabolism
      Feb 28, 2022

      The Natural History of Metachromatic Leukodystrophy

      Recruiting
      • MLD
      • +26 more
      • Palliative Care
      • Hematopoetic Stem Cell Transplantation
      • Pittsburgh, Pennsylvania
        The Program for the Study of Neurodevelopment in Rare Disorders,
      Nov 2, 2021

      GM1 Gangliosidoses, GM2 Gangliosidoses, Tay-Sachs Disease Trial in Minneapolis (miglustat, Ketogenic Diet)

      Terminated
      Has Results
      • GM1 Gangliosidoses
      • +3 more
      • Minneapolis, Minnesota
        University of Minnesota
      Mar 18, 2021

      Tay-Sachs Disease, Sandhoff Disease Trial in Boston, Worcester (AXO-AAV-GM2 Starting Dose, AXO-AAV-GM2 Low Dose, AXO-AAV-GM2

      Recruiting
      • Tay-Sachs Disease
      • Sandhoff Disease
      • AXO-AAV-GM2 Starting Dose
      • +3 more
      • Boston, Massachusetts
      • +1 more
      Jan 26, 2021

      Gangliosidoses, GM1 Gangliosidosis, Morquio B Disease Trial in Hochheim

      Recruiting
      • Gangliosidoses
      • +8 more
        • Hochheim, HE, Germany
          SphinCS Lyso gemeinnützige UG (haftungsbeschränkt)
        Nov 5, 2020

        Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy Trial in Minneapolis (Stem Cell Transplant,

        Completed
        Has Results
        • Adrenoleukodystrophy
        • +10 more
        • Stem Cell Transplant
        • Busulfan, Cyclophosphamide, Antithymocyte Globulin
        • Minneapolis, Minnesota
          Masonic Cancer Center, University of Minnesota
        Dec 3, 2017

        Gene Therapy for Tay-Sachs Disease

        Completed
        • Tay Sachs Disease
        • +2 more
          • Tampa, Florida
          • +1 more
          Dec 3, 2014

          Gangliosidoses, GM2, Sandhoff Disease, Tay-Sachs Disease Trial in Toronto (Pyrimethamine, Leucovorin)

          Completed
          • Gangliosidoses, GM2
          • +2 more
          • Toronto, Ontario, Canada
            The Hospital for Sick Children
          Feb 22, 2012

          GM2 Gangliosidoses, Tay-Sachs, Sandhoff Disease Trial in Washington (Zavesca (Miglustat))

          Completed
          • GM2 Gangliosidoses
          • +2 more
          • Zavesca (Miglustat)
          • Washington, District of Columbia
            Children's National Medical Center
          May 5, 2008