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juvenile Tay-Sachs disease

Showing 1 - 25 of >10,000
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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

      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

      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

      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

      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

        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

        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

        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 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

              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

                Juvenile Systemic Lupus Erythematosus

                Recruiting
                • Systemic Lupus Erythematosus
                • lipid profile
                • Assiut, Egypt
                  Assiut university
                Nov 21, 2023

                Severity in Juvenile and Adult-onset Dermatomyositis

                Not yet recruiting
                • Dermatomyositis
                • evaluation of clinical severity
                • (no location specified)
                Aug 16, 2023

                Fibrinogen to Albumin Ratio and C-reactive Protein to Albumin

                Not yet recruiting
                • Juvenile Idiopathic Arthritis
                • fibrinogen to albumin ratio (AFR) and C-reactive protein to albumin ratio (CAR)
                • (no location specified)
                Aug 29, 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

                Still's Disease, Juvenile Onset, Still's Disease, Adult-Onset Trial in Japan (Anakinra, Placebo)

                Recruiting
                • Still's Disease, Juvenile Onset
                • Still's Disease, Adult-Onset
                • Chiba-shi, Chiba, Japan
                • +17 more
                Apr 3, 2023

                Juvenile Recurrent Respiratory Papillomatosis: Establishment

                Recruiting
                • Juvenile Recurrent Respiratory Papillomatosis
                  • Paris, France
                    hôpital Necker Enfants malades
                  Aug 17, 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

                  Serum Interleukin - 17A in Juvenile Idiopathic Arthritis

                  Not yet recruiting
                  • JIA
                  • serum levels of interleukin-17A in JIA
                  • (no location specified)
                  Jan 15, 2023

                  Autism Spectrum Disorder Trial in Tuscaloosa (STEPS)

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