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Lysosomal 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

    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

    Assess Urine Biomarkers to Predict Nephropathy in Fabry Disease

    Recruiting
    • Fabry Disease
      • Rockville, Maryland
      • +1 more
      Sep 29, 2023

      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

      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 Acid Lipase Activity in Nonalcoholic Fatty Liver

      Recruiting
      • Non-Alcoholic Fatty Liver Disease
      • Peripheral blood withdrawn
      • Rome, Italy
        Department of Translational and Precision Medicine, Sapienza Uni
      Jun 22, 2022

      Krabbe Disease Trial in Buffalo

      Recruiting
      • Krabbe Disease
        • Buffalo, New York
          State University of New York at Buffalo
        Apr 17, 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

        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

          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

            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

            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

            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

              Velaglucerase Alfa Given as Standard Patient Care in Young

              Recruiting
              • Gaucher Disease
              • Standard of Care
              • Fairfax, Virginia
                Lysosomal & Rare Disorders Research & Treatment Center
              Jan 5, 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

              Carotid Structure and Function in MPS Syndromes: A Multicenter

              Completed
              Has Results
              • Mucopolysaccharidoses
                • Orange, California
                • +1 more
                Feb 5, 2021

                Gaucher Disease Trial in Durham, Fairfax (LY3884961, Methylprednisolone, Sirolimus)

                Not yet recruiting
                • Gaucher Disease
                • LY3884961
                • +3 more
                • Durham, North Carolina
                • +1 more
                Aug 1, 2022

                Fabry Disease Trial in Worldwide (migalastat HCl 150 mg)

                Recruiting
                • Fabry Disease
                • migalastat HCl 150 mg
                • Atlanta, Georgia
                • +8 more
                Jan 11, 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

                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

                Fabry Disease Trial in United States (Pegunigalsidase Alfa)

                Available
                • Fabry Disease
                • Pegunigalsidase Alfa
                • Birmingham, Alabama
                • +10 more
                Oct 20, 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

                Fabry Disease Trial in United States (4D-310)

                Recruiting
                • Fabry Disease
                • 4D-310
                • Birmingham, Alabama
                • +5 more
                Mar 24, 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