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Lysosomal Storage Disorder

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

    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

    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

      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

      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

      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

      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

      Bone in Pediatric Gaucher Disease Related to Bone Pathology in

      Recruiting
      • Gaucher Disease
      • Bone Diseases
        • Fairfax, Virginia
          Lysosomal and Rare disorder research and treatment center
        Nov 6, 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

          Complement Activation in the Lysosomal Storage Disorders

          Withdrawn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

          Glycosphingolipid and Glycoprotein Lysosomal Storage Disorders

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

            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

            Autoimmune Hepatitis, Liver Disorder Trial in Monza (Clinical Information)

            Recruiting
            • Autoimmune Hepatitis
            • Liver Disorder
            • Clinical Information
            • Monza, MB, Italy
              Fondazione IRCCS San Gerardo dei Tintori
            Oct 5, 2023

            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

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

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

              Adeno-Associated Virus Antibody Study in Subjects OTC

              Recruiting
              • Ornithine Transcarbamylase Deficiency
              • +2 more
                • Akron, Ohio
                • +2 more
                Jul 22, 2022

                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, 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

                  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

                  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

                  Danon Disease Trial in La Jolla, Philadelphia (RP-A501)

                  Recruiting
                  • Danon Disease
                  • RP-A501
                  • La Jolla, California
                  • +1 more
                  Oct 17, 2023

                  Neurological Manifestations in Gaucher Disease Patients

                  Not yet recruiting
                  • Gaucher Disease
                    • (no location specified)
                    Apr 14, 2023