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gangliosidoses

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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

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

    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

    GM1 and GM2 Gangliosidosis PROspective Neurological Disease

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

      Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients

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

        Gangliosidoses, GM1 Gangliosidosis, Morquio B Disease Trial in Hochheim

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

          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

          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

          Interviews and Video Capture in Patients With GM1 Gangliosidosis

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

          Leukodystrophy, White Matter Disease, Leukoencephalopathies Trial in United States

          Recruiting
          • Leukodystrophy
          • +21 more
            • Palo Alto, California
            • +8 more
            Dec 21, 2021

            GM2 Gangliosidosis Trial in Boston

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

              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

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

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

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

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

                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

                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

                Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy Trial in Minneapolis (Stem Cell Transplant,

                Completed
                Has Results
                • Adrenoleukodystrophy
                • +10 more
                • Stem Cell Transplant
                • Busulfan, Cyclophosphamide, Antithymocyte Globulin
                • Minneapolis, Minnesota
                  Masonic Cancer Center, University of Minnesota
                Dec 3, 2017

                Lysosomal Storage Disorders Diseases in Minority Groups

                Unknown status
                • Lysosomal Storage Diseases
                • Enzyme assay and molecular sequencing
                • Fairfax, Virginia
                  LDRTC
                Jan 18, 2019

                Gangliosidoses GM2 Trial in Toronto (miglustat)

                Completed
                • Gangliosidoses GM2
                • Toronto, Ontario, Canada
                  The Hospital for Sick Children
                May 18, 2016

                Lysosomal Storage Diseases, Inborn Errors of Metabolism Trial in Durham (ALD-601)

                Withdrawn
                • Lysosomal Storage Diseases
                • Inborn Errors of Metabolism
                • ALD-601
                • Durham, North Carolina
                • +1 more
                Jun 27, 2017

                Gene Therapy for Tay-Sachs Disease

                Completed
                • Tay Sachs Disease
                • +2 more
                  • Tampa, Florida
                  • +1 more
                  Dec 3, 2014

                  G(M2) Ganglioside, Tay-Sachs Disease Ganglioside, Sandhoff Disease Ganglioside Trial in New York, Cleveland, Toronto

                  Withdrawn
                  • G(M2) Ganglioside
                  • +2 more
                  • New York, New York
                  • +2 more
                  Feb 21, 2013