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Lysosomal Storage Diseases

Showing 1 - 25 of >10,000
STUDY
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Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV A Trial in San Francisco (There is no intervention)

Recruiting
  • Mucopolysaccharidosis I
  • +7 more
  • There is no intervention
  • San Francisco, California
    University of California San Francisco
Nov 9, 2022

Genetic Studies of Lysosomal Storage Disorders

Enrolling by invitation
  • Lysosomal Storage Disorders
  • +2 more
    • Bethesda, Maryland
      National Institutes of Health Clinical Center
    Jan 21, 2023

    Pompe Disease, Muscle Loss, Obesity Trial (Multi-ingredient supplement (PDT-MIS), Placebo (PLA))

    Not yet recruiting
    • Pompe Disease
    • +7 more
    • Multi-ingredient supplement (PDT-MIS)
    • Placebo (PLA)
    • (no location specified)
    Nov 8, 2023

    Lysosomal Storage Diseases, Metachromatic Leukodystrophy Trial in Milan (OTL-200)

    Recruiting
    • Lysosomal Storage Diseases
    • Metachromatic Leukodystrophy
    • OTL-200
    • Milan, Italy
      Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR
    Nov 16, 2022

    Inborn Errors of Cholesterol Synthesis and Related Disorders

    Recruiting
    • Lysosomal Storage Disease
    • Cholesterol Metabolism
      • Bethesda, Maryland
        National Institutes of Health Clinical Center
      Dec 28, 2022

      Lysosomal Storage Disease, Metachromatic Leukodystrophy Trial in Milan (OTL-200 Gene Therapy)

      Active, not recruiting
      • Lysosomal Storage Disease
      • Metachromatic Leukodystrophy
      • OTL-200 Gene Therapy
      • Milan, Italy
        Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR
      Nov 4, 2022

      MPS I, MPS II, MPS IVA Trial in San Francisco (Aldurazyme (laronidase))

      Recruiting
      • MPS I
      • +8 more
      • Aldurazyme (laronidase)
      • San Francisco, California
        University of California
      Apr 21, 2022

      Complement Activation in the Lysosomal Storage Disorders

      Withdrawn
      • Fabry Disease
      • +3 more
      • Complement measurements
      • Melbourne, Victoria, Australia
        The Royal Melbourne Hospital
      Jun 2, 2021

      Molecular and Cellular Mechanisms of Lysosomal Storage Diseases

      Unknown status
      • Lysosomal Storage Disorders
        • Fairfax, Virginia
          Lysosomal and Rare Disorders Research and Treatment Center, Inc
        Feb 22, 2021

        Lysosomal Storage Disease, Metachromatic Leukodystrophy Trial in Milan (OTL-200)

        Active, not recruiting
        • Lysosomal Storage Disease
        • Metachromatic Leukodystrophy
        • OTL-200
        • Milan, Italy
          Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR
        Sep 29, 2021

        Pompe Disease, Glycogen Storage Disease Type II, Lysosomal Storage Diseases Trial in Gainesville (Diet and Exercise)

        Completed
        • Pompe Disease
        • +2 more
        • Diet and Exercise
        • Gainesville, Florida
          University of Florida Clinical Research Center
        May 13, 2021

        Fabry Disease, Fabry Disease, Cardiac Variant, Lysosomal Storage Diseases Trial in Wuerzburg (blood sampling

        Enrolling by invitation
        • Fabry Disease
        • +4 more
        • blood sampling (alpha-Galactosidase & LysoGb3)
        • Wuerzburg, Bavaria, Germany
          University Hospital Wuerzburg
        Jul 2, 2021

        Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2 Trial in Worldwide (SPK-3006)

        Active, not recruiting
        • Pompe Disease
        • +6 more
        • SPK-3006
        • Phoenix, Arizona
        • +28 more
        Jul 22, 2022

        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

        Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease

        Completed
        • Lysosomal Storage Diseases
        • +2 more
          • Tirana, Albania
          • +7 more
          May 27, 2021

          Fabry Disease, Lysosomal Storage Diseases Trial in Worldwide (FLT190)

          Unknown status
          • Fabry Disease
          • Lysosomal Storage Diseases
          • FLT190
          • Berlin, Germany
          • +4 more
          Feb 2, 2021

          Biomarker for Maroteaux-Lamy Disease (BioMaroteaux)

          Active, not recruiting
          • Lysosomal Storage Disease
          • +4 more
            • Cairo, Egypt
            • +4 more
            May 17, 2021

            Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2 Trial in Worldwide (Neutralizing Antibody to SPK-3006

            Completed
            • Pompe Disease
            • +5 more
            • Neutralizing Antibody to SPK-3006 capsid
            • Phoenix, Arizona
            • +17 more
            Jul 14, 2021

            Fabry Disease, Lysosomal Storage Diseases Trial in London (FLT190)

            Recruiting
            • Fabry Disease
            • Lysosomal Storage Diseases
            • FLT190
            • London, United Kingdom
              Royal Free London
            Sep 18, 2020

            Children With Metachromatic Leukodystrophy

            Terminated
            • Lipid Metabolism Disorders
            • +13 more
              • Torrance, California
              • +13 more
              Mar 16, 2021

              Hurler Syndrome, Inherited Metabolic Disorder, Lysosomal Storage Disorder Trial in Los Angeles, Palo Alto (rivogenlecleucel,

              No longer available
              • Hurler Syndrome
              • +4 more
              • Los Angeles, California
              • +1 more
              Oct 1, 2020

              Glycosphingolipid and Glycoprotein Lysosomal Storage Disorders

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

                Carbon-13 Magnetic Resonance Spectroscopy in Glycogen Storage

                Recruiting
                • Glycogen Storage Disease
                • +2 more
                • No intervention
                • Copenhagen, Copenhange, Denmark
                  Neuromuscular Research Unit, 8077
                Oct 26, 2022

                Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease Trial in Gainesville, Decatur, Detroit

                Completed
                Has Results
                • Pompe Disease
                • +2 more
                • alglucosidase alfa
                • Gainesville, Florida
                • +2 more
                Jul 29, 2022

                Baby Detect : Genomic Newborn Screening

                Recruiting
                • Congenital Adrenal Hyperplasia
                • +126 more
                  • Liege, Wallonia, Belgium
                    CRMN, Hôpital La Citadelle
                  Jan 24, 2023