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Tay Sachs

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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

      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

      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

        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

        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

        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

        GM2 Gangliosidosis Trial in Boston

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

          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

            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

            Glycosphingolipid and Glycoprotein Lysosomal Storage Disorders

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

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

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

                Leukodystrophy: An Observational Study at Sohag University

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

                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

                Autism, Autism Spectrum Disorder Trial in Plainwell (Work Chat: An Interactive Virtual Workday)

                Enrolling by invitation
                • Autism
                • Autism Spectrum Disorder
                • Work Chat: An Interactive Virtual Workday
                • Plainwell, Michigan
                  Michigan Career and Technical Institute
                Jan 27, 2023

                Autism Spectrum Disorder Trial in Tuscaloosa (STEPS)

                Recruiting
                • Autism Spectrum Disorder
                • STEPS
                • Tuscaloosa, Alabama
                  Center for Youth Development and Intervention
                Oct 11, 2022

                Other Instability, Shoulder, Hill-Sachs Lesion Trial in Winnipeg, Ottawa (Bankart repair and remplissage, Bankart repair)

                Active, not recruiting
                • Other Instability, Shoulder
                • Hill-Sachs Lesion
                • Bankart repair and remplissage
                • Bankart repair
                • Winnipeg, Manitoba, Canada
                • +1 more
                Apr 19, 2022

                Autism Spectrum Disorder Trial (Usual care, 'Got Transition' toolkit and resources, PSE intervention)

                Withdrawn
                • Autism Spectrum Disorder
                • Usual care
                • +2 more
                • (no location specified)
                Apr 15, 2022

                Substance Use Disorders Trial in Newton (Advanced Emotion App, Basic Emotion App)

                Completed
                • Substance Use Disorders
                • Advanced Emotion App
                • Basic Emotion App
                • Newton, Massachusetts
                  Lisa Najavits
                Apr 25, 2022

                Shoulder Dislocation Closed Traumatic Trial in Bologna (fresh-frozen humeral head osteochondral allograft reconstruction for

                Recruiting
                • Shoulder Dislocation Closed Traumatic
                • fresh-frozen humeral head osteochondral allograft reconstruction for reverse Hill-Sachs lesion
                • Bologna, Italy
                  IRCCS Istituto Ortopedico Rizzoli
                Mar 30, 2022

                Autism Spectrum Disorder Trial in San Diego (TAY SUCCESS, Usual Care)

                Completed
                • Autism Spectrum Disorder
                • TAY SUCCESS
                • Usual Care
                • San Diego, California
                  Rady Children's Hospital San Diego
                Jun 28, 2021