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infantile GM2 gangliosidosis

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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

    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

    Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients

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

      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

      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

        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

            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

            Gangliosidoses, GM1 Gangliosidosis, Morquio B Disease Trial in Hochheim

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

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

              Recruiting
              • GM1 Gangliosidosis
              • LYS-GM101
              • Orange, California
              • +2 more
              Jun 30, 2021

              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

                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

                Human Milk Oligosaccharides and Microbiomes on Infantile Colic

                Recruiting
                • Infantile Colic
                • +3 more
                  • Tainan, Taiwan
                    National Cheng Kung University & Hospital
                  Aug 7, 2023

                  Leukodystrophy: An Observational Study at Sohag University

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

                  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

                    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

                    Infantile Nystagmus Syndrome Trial in Zagazig (Graded Anderson procedure, Kestenbaum procedure)

                    Active, not recruiting
                    • Infantile Nystagmus Syndrome
                    • Graded Anderson procedure
                    • Kestenbaum procedure
                    • Zagazig, Egypt
                      Zagazig University
                    Jul 12, 2023