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Sandhoff

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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

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

    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

    GM1 and GM2 Gangliosidosis PROspective Neurological Disease

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

      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

      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

      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

          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

          Ewing Sarcoma Trial in Warsaw (Naxitamab)

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

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

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

            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

            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

            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

              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

              Stroke, Hemiplegia Trial in San Diego (Theraband with exercise video, usual care with advice to exercise)

              Completed
              • Stroke
              • Hemiplegia
              • Theraband with exercise video
              • usual care with advice to exercise
              • San Diego, California
                Kaiser Permanente
              Aug 21, 2018

              Lysosomal Storage Disease, Peroxisomal Disorder Trial in Minneapolis (drug, radiation, biological)

              Completed
              Has Results
              • Lysosomal Storage Disease
              • Peroxisomal Disorder
              • Minneapolis, Minnesota
                Masonic Cancer Center, University of Minnesota
              Dec 3, 2017

              Lysosomal Storage Diseases, Inborn Errors of Metabolism Trial in Durham (ALD-601)

              Withdrawn
              • Lysosomal Storage Diseases
              • Inborn Errors of Metabolism
              • ALD-601
              • Durham, North Carolina
              • +1 more
              Jun 27, 2017

              Gangliosidoses GM2 Trial in Toronto (miglustat)

              Completed
              • Gangliosidoses GM2
              • Toronto, Ontario, Canada
                The Hospital for Sick Children
              May 18, 2016

              Phosphoglycerate Kinase (PGK) Deficiency Trial in Nashville (Unrelated Donor BMT)

              Completed
              • Phosphoglycerate Kinase (PGK) Deficiency
              • Unrelated Donor BMT
              • Nashville, Tennessee
                Vanderbilt Children's Hospital
              Nov 22, 2016

              Peripheral Arterial Disease Trial in Porto Alegre (Device: treadmill, Device: treadmill Train_Comb_aerobic)

              Unknown status
              • Peripheral Arterial Disease
              • Device: treadmill
              • Device: treadmill Train_Comb_aerobic
              • Porto Alegre, Rio Grande do Sul, Brazil
                Hospital De Clínicas de Porto Alegre
              Mar 31, 2016

              Gene Therapy for Tay-Sachs Disease

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