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Sandhoff Disease

Showing 1 - 25 of >10,000
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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

    GM1 and GM2 Gangliosidosis PROspective Neurological Disease

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

      GM2 Gangliosidosis, Supportive Care Trial in Kashan, Mashhad, Tehran (Miglustat)

      Recruiting
      • GM2 Gangliosidosis
      • Supportive Care
      • Kashan, Isfahan, Iran, Islamic Republic of
      • +2 more
      Oct 23, 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

      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

      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

      Gangliosidoses, GM1 Gangliosidosis, Morquio B Disease Trial in Hochheim

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

        Tay-Sachs Disease Sandhoff Disease Trial in Worldwide (venglustat GZ402671, )

        Active, not recruiting
        • Tay-Sachs Disease Sandhoff Disease
        • venglustat GZ402671
        • placebo
        • Los Angeles, California
        • +23 more
        Jan 31, 2022

        Glycosphingolipid and Glycoprotein Lysosomal Storage Disorders

        Recruiting
        • Neurological Regression
        • +3 more
          • Bethesda, Maryland
            National Institutes of Health Clinical Center
          Jan 14, 2023

          Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy Trial in Minneapolis (drug, procedure,

          Completed
          Has Results
          • Adrenoleukodystrophy
          • +8 more
          • Minneapolis, Minnesota
            Masonic Cancer Center, University of Minnesota
          Jul 10, 2019

          Infantile GM2 Gangliosidosis (Disorder) Trial in Kingston (TSHA-101)

          Active, not recruiting
          • Infantile GM2 Gangliosidosis (Disorder)
          • TSHA-101
          • Kingston, Ontario, Canada
            Queen's University/Kingston Health Sciences Centre
          Sep 12, 2022

          Biomarker for Gangliosidosis: BioGM1/BioGM2 (BioGM1/GM2)

          Active, not recruiting
          • Hepato-splenomegaly
          • +3 more
            • Cairo, Egypt
            • +4 more
            May 12, 2021

            Ewing Sarcoma Trial in Warsaw (Naxitamab)

            Not yet recruiting
            • Ewing Sarcoma
            • Warsaw, Mazowian, Poland
              Mother and Child Institute
            Jul 28, 2023

            Peripheral Artery Disease Trial in Aurora (Intervention A - Vascular Care + site 6MWT administration, Intervention B - Standard

            Recruiting
            • Peripheral Artery Disease
            • Intervention A - Vascular Care + site 6MWT administration
            • +3 more
            • Aurora, Colorado
              University of Colorado Anschutz Medical Campus
            Jul 22, 2021

            GM2 Gangliosidosis Trial in Boston

            Recruiting
            • GM2 Gangliosidosis
              • Boston, Massachusetts
                Massachusetts General Hospital
              Sep 8, 2020

              Diabetes, Type 2 Trial in Hong Kong (Simvastatin 10mg, Ezetimibe 10mg)

              Enrolling by invitation
              • Diabetes Mellitus, Type 2
              • Simvastatin 10mg
              • Ezetimibe 10mg
              • Hong Kong, Hong Kong
                Diabetes Centre, 2/F, Block L, Department of Medicine, Queen Mar
              Nov 21, 2022

              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

              Pompe Disease Trial in New York, Fairfax (biological, drug, other)

              Not yet recruiting
              • Pompe Disease
              • Cipaglucosidase alfa
              • +3 more
              • New York, New York
              • +1 more
              Nov 2, 2023

              Chronic Rhinosinusitis Associated With Inflammatory Bowel

              Not yet recruiting
              • Intestinal Inflammation
              • Rhinosinusitis
              • Observational study
              • (no location specified)
              Nov 21, 2023

              Oral Lichen Planus, Topical Sulfasalazine, Corticosteroids Trial in Cairo (sulfasalazine 500 MG, corticosteroids)

              Active, not recruiting
              • Oral Lichen Planus
              • +2 more
              • Cairo, Egypt
                Faculty of Dentistry, Cairo University
              Sep 23, 2023

              COrSIcA: Disease Measurement

              Not yet recruiting
              • Anal Intraepithelial Neoplasia
              • +2 more
              • Physical examination
              • (no location specified)
              May 4, 2023

              Crohn's Disease Trial (MR Enterography (MRE))

              Not yet recruiting
              • Crohn's Disease
              • MR Enterography (MRE)
              • (no location specified)
              Nov 13, 2023

              Gastroesophageal Reflux Disease Trial in Assiut (Nursing Teaching)

              Not yet recruiting
              • Gastroesophageal Reflux Disease
              • Nursing Teaching
              • Assiut, Egypt
                Faculty of Nursing Assiut University
              Jul 10, 2023

              Patients With Von Hippel Lindau Disease

              Not yet recruiting
              • Von Hippel Lindau Disease
                • Houston, Texas
                  M D Anderson Cancer Center
                Jul 18, 2023