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lysosomal storage disease

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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 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

    Biomarker for Maroteaux-Lamy Disease (BioMaroteaux)

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

      Lysosomal Storage Disease, Peroxisomal Disorder Trial in Minneapolis (drug, radiation, biological)

      Completed
      Has Results
      • Lysosomal Storage Disease
      • Peroxisomal Disorder
      • Minneapolis, Minnesota
        Masonic Cancer Center, University of Minnesota
      Dec 3, 2017

      The Classification and Cause of Leukodystrophies of Unknown

      Completed
      • Lysosomal Storage Disease
        • San Francisco, California
        • +4 more
        Jun 30, 2017

        Cognitive Decline, Mucopolysaccharidosis I, Hurler-Scheie Syndrome Trial in Oakland, Torrance, Minneapolis (laronidase)

        Completed
        • Cognitive Decline
        • +4 more
        • Oakland, California
        • +2 more
        Jan 22, 2016

        Non Malignant Disorders, Immunodeficiencies, Congenital Marrow Failures Trial in Durham (Unrelated Umbilical Cord Blood

        Completed
        Has Results
        • Non Malignant Disorders
        • +7 more
        • Unrelated Umbilical Cord Blood Transplant
        • Reduced Intensity Conditioning
        • Durham, North Carolina
          Duke University Medical Center Pediatric Blood and Marrow Transp
        Jul 23, 2014

        Spinal Cord Compression, Mucopolysaccharidosis I, Hurler-Scheie Syndrome Trial in Torrance, Helsinki (laronidase)

        Terminated
        • Spinal Cord Compression
        • +4 more
        • Torrance, California
        • +1 more
        Feb 19, 2013

        Magnetic Stimulation of the Human Nervous System

        Completed
        • Demyelinating Disease
        • +4 more
          • Bethesda, Maryland
            National Institute of Neurological Disorders and Stroke (NINDS)
          Mar 3, 2008