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Inherited Metabolic Disorders

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Inherited Diseases, Caregiving, and Social Networks

Completed
  • Undiagnosed Disease
  • +4 more
    • Bethesda, Maryland
      National Institutes of Health Clinical Center
    Jan 6, 2023

    Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome Trial in Minneapolis (Stem Cell Transplantation, IMD

    Recruiting
    • Mucopolysaccharidosis Disorders
    • +28 more
    • Stem Cell Transplantation
    • +7 more
    • Minneapolis, Minnesota
      Masonic Cancer Center, University of Minnesota
    Nov 1, 2022

    Caregiving Networks Across Disease Context and the Life Course

    Recruiting
    • Inherited Metabolic Disorders
    • +4 more
      • Bethesda, Maryland
        National Institutes of Health Clinical Center
      Aug 3, 2022

      Primary Immune Deficiency Disorders, Metabolic Disease Trial in Saint Petersburg (Haploidentical Hematopoietic Cell

      Recruiting
      • Primary Immune Deficiency Disorders
      • Metabolic Disease
      • Haploidentical Hematopoietic Cell Transplantation
      • Saint Petersburg, Florida
        Johns Hopkins All Children's Hospital
      Sep 9, 2021

      AML, ALL, MDS Trial in New York (Clofarabine, Fludarabine, Busulfan)

      Recruiting
      • AML
      • +8 more
      • New York, New York
        Memorial Sloan Kettering Cancer Center
      May 13, 2022

      Inherited Metabolic Disorder of Nervous System, Schizophrenia Trial in Grenoble (Sulfitest)

      Recruiting
      • Inherited Metabolic Disorder of Nervous System
      • Schizophrenia
      • Sulfitest
      • Grenoble, France
        CHU Grenoble Alpes
      May 6, 2022

      Sickle Cell Disease, Thalassemia, High Risk Hematologic Disorders Trial in Minneapolis (Blood and Marrow Transplant)

      Recruiting
      • Sickle Cell Disease
      • +4 more
      • Blood and Marrow Transplant
      • Minneapolis, Minnesota
        Masonic Caner Center at University of Minnesota
      May 23, 2022

      Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

      Recruiting
      • Primary Immunodeficiency (PID)
      • +4 more
      • data collection
      • Pittsburgh, Pennsylvania
        UPMC Children's Hospital of Pittsburgh
      Feb 9, 2022

      Inherited Metabolic Disorders Who Underwent Hematopoietic Stem

      Terminated
      • Inherited Metabolic Disorders (IMD)
      • Safety and efficacy assessments
      • Minneapolis, Minnesota
        University of Minnesota
      Mar 10, 2021

      Incidence of Metabolic Disorders in Neonatal Convulsions

      Not yet recruiting
      • Neonatal Convulsions
      • MRI
      • (no location specified)
      Nov 6, 2023

      Metachromatic Leukodystrophy Trial in Durham (Enriched Hematopoetic Stem Cell Infusion)

      No longer available
      • Metachromatic Leukodystrophy
      • Enriched Hematopoetic Stem Cell Infusion
      • Durham, North Carolina
        Duke University
      Oct 6, 2020

      Adrenoleukodystrophy, Batten Disease, Mucopolysaccharidosis II Trial in Durham (DUOC-01)

      Recruiting
      • Adrenoleukodystrophy
      • +11 more
      • DUOC-01
      • Durham, North Carolina
        Duke University Medical Center
      Oct 24, 2022

      Orphan Drugs for Inherited Metabolic Diseases

      Recruiting
      • Inborn Errors of Metabolism
        • Lausanne, Vaud, Switzerland
          Lausanne University Hospital
        Apr 19, 2023

        The Molecular Basis of Inherited Reproductive Disorders

        Recruiting
        • Hypogonadotropic Hypogonadism
        • +3 more
          • Boston, Massachusetts
            Massachusetts General Hospital
          Jul 31, 2023

          Studies of Inherited Diseases of Metabolism

          Completed
          • Multiple Endocrine Neoplasia
            • Bethesda, Maryland
              National Institutes of Health Clinical Center
            Jan 19, 2023

            Cancer in Inherited Bone Marrow Failure Syndromes

            Recruiting
            • Diamond Blackfan Anemia
            • +4 more
              • Bethesda, Maryland
              • +1 more
              Jan 24, 2023

              Biomarker Research in Inherited Movement Disorders

              Recruiting
              • Inherited Movement Disorders
              • +2 more
              • Clinical follow-up
              • Paris, France
                Hôpital Pitié Salpetrière
              Aug 11, 2022

              Inherited Reproductive Disorders

              Recruiting
              • Genetic Disorder
              • +3 more
                • Bethesda, Maryland
                • +1 more
                Dec 29, 2022

                Study of Inherited Neurological Disorders

                Recruiting
                • Motor Neuron Disease
                • +3 more
                  • Bethesda, Maryland
                    National Institutes of Health Clinical Center
                  Jan 26, 2023

                  Adaptive Optics Retinal Imaging in Inherited and Acquired

                  Recruiting
                  • Genetic Disease
                  • Inherited Disease
                  • Adaptive Optics Retinal Camera
                  • Toronto, Ontario, Canada
                    The Hospital for Sick Children
                  Jul 27, 2022

                  Rare Inherited Bleedig Disorders in Children at Sohag University

                  Recruiting
                  • Rare Inherited Bleeding Disorders in Children at Sohag University Hospital
                  • blood laboratory investigation(coagulation profile- platelet function test)
                  • Sohag, Egypt
                    Sohag University Hospital
                  May 15, 2022

                  Von Hippel-Lindau: Clinical Manifestations, Diagnosis,

                  Recruiting
                  • Kidney Cancer
                  • +4 more
                    • Bethesda, Maryland
                      National Institutes of Health Clinical Center
                    Jan 25, 2023

                    Cardiac Manifestation and Inherited Metabolic Diseases

                    Recruiting
                    • Metabolism, Inborn Errors
                      • Lausanne, Vaud, Switzerland
                        Lausanne University Hospital
                      Aug 7, 2021

                      Acquired and Inherited Bone Marrow Failure Syndromes

                      Recruiting
                      • Severe Aplastic Anemia
                      • +2 more
                        • Bethesda, Maryland
                          National Institutes of Health Clinical Center
                        Aug 12, 2022

                        Stem Cell Transplantation Trial in Houston (Fludarabine, Dexamethasone, Cyclophosphamide)

                        Not yet recruiting
                        • Stem Cell Transplantation
                        • Houston, Texas
                          M D Anderson Cancer Center
                        May 4, 2022