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lysosomal storage disease

Showing 1 - 25 of >10,000
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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

    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

      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

      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 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

      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

      Pompe Disease Trial in New York, Fairfax (biological, drug, other)

      Not yet recruiting
      • Pompe Disease
      • Cipaglucosidase alfa
      • +3 more
      • New York, New York
      • +1 more
      Nov 2, 2023

      Complement Activation in the Lysosomal Storage Disorders

      Withdrawn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

      Late-Onset Pompe Disease, Lysosomal Disease Trial in Durham (IMT therapy using the Pr02 mobile device)

      Recruiting
      • Late-Onset Pompe Disease
      • Lysosomal Disease
      • IMT therapy using the Pr02 mobile device
      • Durham, North Carolina
        Duke University Medical Center
      Jul 21, 2022

      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 (SPK-3006)

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

        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

        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

        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

          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 Worldwide (FLT190)

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

          Lysosomal Acid Lipase Deficiency, Cholesterol Ester Storage Disease, Wolman Disease Trial in Worldwide

          Recruiting
          • Lysosomal Acid Lipase Deficiency
          • +6 more
            • Stanford, California
            • +27 more
            Jan 11, 2021

            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

            Biospecimen in Pediatric Patients and Adult Guardians Diagnosed

            Recruiting
            • Glycogen Storage Disease Type IB
            • Specimen Donation
            • Woburn, Massachusetts
              Sanguine BioSciences, Inc.
            Jun 22, 2023

            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

              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

              Baby Detect : Genomic Newborn Screening

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