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infantile Tay-Sachs disease

Showing 1 - 25 of >10,000
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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

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

GM1 and GM2 Gangliosidosis PROspective Neurological Disease

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

      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

      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 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

      Caregiving Networks Across Disease Context and the Life Course

      Recruiting
      • Inherited Metabolic Disorders
      • +4 more
        • Bethesda, Maryland
          National Institutes of Health Clinical Center
        Aug 3, 2022

        GM2 Gangliosidosis, Niemann-Pick Disease, Type C Trial in Curitiba, Porto Alegre, Rio De Janeiro (AZ-3102 (Dose 1), Placebo,

        Not yet recruiting
        • GM2 Gangliosidosis
        • Niemann-Pick Disease, Type C
        • AZ-3102 (Dose 1)
        • +2 more
        • Curitiba, Brazil
        • +2 more
        Feb 24, 2023

        GM2 Gangliosidosis Trial in Boston

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

          Gangliosidoses, GM1 Gangliosidosis, Morquio B Disease Trial in Hochheim

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

            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

            Glycosphingolipid and Glycoprotein Lysosomal Storage Disorders

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

              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

              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

                Leukodystrophy: An Observational Study at Sohag University

                Not yet recruiting
                • Children With Leukodystrophy
                • MRI
                • (no location specified)
                Mar 2, 2021

                Krabbe Disease Trial in Ann Arbor (FBX-101)

                Recruiting
                • Krabbe Disease
                • FBX-101
                • Ann Arbor, Michigan
                  University of Michigan Hospitals - Michigan Medicine
                Feb 13, 2023

                Mental Health Wellness 1, Mental Health Wellness 2, Occupational Problems Trial (iCareer Soft Skills Training for Employment)

                Not yet recruiting
                • Mental Health Wellness 1
                • +2 more
                • iCareer Soft Skills Training for Employment
                • (no location specified)
                Mar 16, 2023

                Pompe Disease Infantile-Onset Trial in China (GC301)

                Not yet recruiting
                • Pompe Disease Infantile-Onset
                • GC301
                • Beijing, China
                • +4 more
                Apr 2, 2023

                Infantile Spasm Trial in Randwick, Singapore (Tricaprilin)

                Recruiting
                • Infantile Spasm
                • Randwick, New South Wales, Australia
                • +3 more
                Dec 13, 2022

                Hemangioma Trial in Chengdu

                Recruiting
                • Hemangioma
                  • Chengdu, Sichuan, China
                    West China Hospital of Sichuan University
                  Jan 15, 2023

                  Spontaneous Evisceration of Infantile Umbilical Hernia

                  Not yet recruiting
                  • Pediatric Disorder
                  • Exploration
                  • Assiut, Egypt
                    Ahmed Ali
                  May 2, 2023

                  Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients

                  Recruiting
                  • GM1 Gangliosidosis
                    • Oakland, California
                    • +5 more
                    Feb 2, 2023

                    Infantile-onset Pompe Disease Trial in Beijing (Genetic: GC301)

                    Recruiting
                    • Infantile-onset Pompe Disease
                    • Genetic: GC301
                    • Beijing, Beijing, China
                      Bayi Children's Hospital, Seventh Medical Center, PLA general ho
                    Sep 30, 2022