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juvenile GM2 gangliosidosis

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GM1 and GM2 Gangliosidosis PROspective Neurological Disease

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

    Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients

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

      Lysosomal Diseases, Gangliosidosis, GM1 Trial run by the NHGRI (biological, drug, diagnostic test, other, procedure, behavioral)

      Recruiting
      • Lysosomal Diseases
      • +2 more
      • AAV9-GLB1
      • +20 more
      • Bethesda, Maryland
        National Institutes of Health Clinical Center
      Jan 19, 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

      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

        Interviews and Video Capture in Patients With GM1 Gangliosidosis

        Recruiting
        • GM1 Gangliosidosis
        • Natural history
        • Plymouth, Massachusetts
          Casimir Trials
        Aug 23, 2021

        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

        GM1 Gangliosidosis, GM1 Gangliosidosis, Type I, GM1 Gangliosidosis, Type 2 Trial in Worldwide (PBGM01)

        Recruiting
        • GM1 Gangliosidosis
        • +3 more
        • PBGM01
        • Oakland, California
        • +9 more
        Mar 10, 2022

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

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

          Glycosphingolipid and Glycoprotein Lysosomal Storage Disorders

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

            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

              GM2 Gangliosidosis Trial in Boston

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

                ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn

                Enrolling by invitation
                • Acid Sphingomyelinase Deficiency
                • +13 more
                • Confirmatory Testing
                • Bronx, New York
                • +9 more
                May 9, 2022

                GM1 Gangliosidosis Trial in Orange, Paris, Manchester (LYS-GM101)

                Recruiting
                • GM1 Gangliosidosis
                • LYS-GM101
                • Orange, California
                • +2 more
                Jun 30, 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

                Myelomonocytic Leukemia Trial in United States (Ruxolitinib)

                Completed
                Has Results
                • Myelomonocytic Leukemia
                • Tampa, Florida
                • +5 more
                Sep 9, 2022

                Juvenile Idiopathic Arthritis Trial in Izmir (Inspiratory muscle training (IMT))

                Enrolling by invitation
                • Juvenile Idiopathic Arthritis
                • Inspiratory muscle training (IMT)
                • İzmir, Turkey
                  Izmir Katip Celebi University
                Jul 29, 2022

                Opioid Use, Substance Use Trial in United States (Trust-based Relational Intervention Group Training, Trust-based Relational

                Recruiting
                • Opioid Use
                • Substance Use
                • Trust-based Relational Intervention Training
                • +2 more
                • Chicago, Illinois
                • +11 more
                Jan 20, 2023

                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

                Juvenile Idiopathic Arthritis Trial in Al Kharj (Aqua-Plyometric Exercise, Standard physical therapy)

                Completed
                • Juvenile Idiopathic Arthritis
                • Aqua-Plyometric Exercise
                • Standard physical therapy
                • Al Kharj, Riyadh, Saudi Arabia
                  Ragab K. Elnaggar
                Mar 22, 2022

                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