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infantile GM1 gangliosidosis

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Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients

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

    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 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

      Interviews and Video Capture in Patients With GM1 Gangliosidosis

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

      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

      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

      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

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

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

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

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

          Brain Metastases, Radiotherapy Side Effect, Cognitive Impairment Trial in Guangzhou (Monosialotetrahexosyl ganglioside (GM1),

          Recruiting
          • Brain Metastases
          • +4 more
          • Monosialotetrahexosyl ganglioside (GM1)
          • Control
          • Guangzhou, Guangdong, China
            Sun Yat-sen University Cancer Center
          Nov 3, 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

              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

              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

              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

              Leukodystrophy: An Observational Study at Sohag University

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

              Human Milk Oligosaccharides and Microbiomes on Infantile Colic

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

                To Evaluate the Effectiveness of GM1 in the Prevention of Albumin-paclitaxel Therapy Induced Peripheral Neuropathy in Breast

                Not yet recruiting
                • To Evaluate the Effectiveness of GM1 in the Prevention of Albumin-paclitaxel Therapy Induced Peripheral Neuropathy in Breast Cancer Patients
                • Monosialotetrahexose ganglioside sodium injection
                • (no location specified)
                Mar 27, 2023

                Anatomic Stage IV Breast Cancer AJCC v8, Chemo-Induced Peripheral Neuropathy, Metastatic Breast Carcinoma Trial (Paclitaxel,

                Not yet recruiting
                • Anatomic Stage IV Breast Cancer AJCC v8
                • +2 more
                • (no location specified)
                Feb 21, 2023

                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

                Infantile Spasm Trial in Randwick, Singapore (Tricaprilin)

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