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mucolipidosis

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Mucolipidosis Type IV Natural History Study

Recruiting
  • Mucolipidosis Type IV
    • Boston, Massachusetts
      Massachusetts General Hospital
    Mar 13, 2023

    Biomarker for Mucolipidosis Disorder Type I, II, III, IV (BioML)

    Active, not recruiting
    • Skeletal Abnormalities
    • Psychomotor Retardation
      • Cairo, Egypt
      • +4 more
      May 12, 2021

      Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence Trial in Sioux Falls, Sydney

      Recruiting
      • Rare Disorders
      • +309 more
        • Sioux Falls, South Dakota
        • +1 more
        Jun 20, 2022

        The Natural History of Mucolipidosis Type IV

        Unknown status
        • Mucolipidosis Type IV
          • Dallas, Texas
            Baylor Institute of Metabolic Disease
          Jul 17, 2018

          Longitudinal Studies of the Glycoproteinoses

          Unknown status
          • Aspartylglucosaminuria
          • +8 more
            • North Charleston, South Carolina
              Greenwood Genetic Center
            Jul 31, 2017

            The Natural History and Pathogenesis of Mucolipidosis Type IV

            Completed
            • Mucolipidosis Type IV
              • Bethesda, Maryland
                National Institutes of Health Clinical Center, 9000 Rockville Pi
              Jun 30, 2017

              Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Mannosidosis Trial in Minneapolis (Stem Cell Transplant, Busulfan,

              Completed
              Has Results
              • Mucopolysaccharidosis I
              • +3 more
              • Stem Cell Transplant
              • Busulfan, Cyclophosphamide, ATG
              • Minneapolis, Minnesota
                Masonic Cancer Center, University of Minnesota
              Dec 3, 2017

              Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy Trial in Minneapolis (drug, procedure,

              Completed
              Has Results
              • Adrenoleukodystrophy
              • +8 more
              • Minneapolis, Minnesota
                Masonic Cancer Center, University of Minnesota
              Jul 10, 2019

              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

              Disease Progression in Patients With Mucopolysaccharidosis IVA

              Terminated
              • Mucopolysaccharidosis Type IVA
                • Decatur, Georgia
                  Emory University, Dept of Human Genetics
                Sep 2, 2014