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inherited metabolic disease

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STUDY
CONDITIONS
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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

    Cardiac Manifestation and Inherited Metabolic Diseases

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

      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

      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

      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

      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

        Ultra-rare Inherited Metabolic and Degenerative Neurological

        Recruiting
        • Inherited Disease
        • +5 more
        • collection of data
        • Milano, Italy
          Fondazione IRCCS Istituto Neurologico Carlo Besta
        May 6, 2021

        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

        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 Disorders Who Underwent Hematopoietic Stem

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

        Orphan Drugs for Inherited Metabolic Diseases

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

          Studies of Inherited Diseases of Metabolism

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

            Global Patient Registry of Inherited Retinal Diseases

            Recruiting
            • Inherited Retinal Diseases
            • Standard of Care
            • Birmingham, Alabama
            • +1 more
            Jul 21, 2023

            Families With Inherited Eye Disease

            Recruiting
            • Inherited Eye Disease
              • La Jolla, California
              • +5 more
              Aug 20, 2022

              Pattern of Metabolic Causes of Neonatal Hypoglycemia

              Not yet recruiting
              • Neonatal Hypoglycemia
              • Metabolic Disease
                • (no location specified)
                Jul 24, 2023

                Inherited Cardiac cONditions In Kids

                Not yet recruiting
                • Inherited Cardiac Conditions
                • Cardiovascular Diseases
                • Patients with inherited cardiac condition, onset <16 years old and Parents
                • (no location specified)
                Aug 10, 2022

                Study of Inherited Neurological Disorders

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

                  Inherited Metabolic Diseases Requiring Restrictive and Specific Diet Trial in Marseille (data assessment of the state of health

                  Recruiting
                  • Inherited Metabolic Diseases Requiring Restrictive and Specific Diet
                  • data assessment of the state of health and quality of life of patients and their families
                  • Marseille, France
                    Hôpital de la Timone Assistance Publique Hôpitaux de Marseille
                  Sep 10, 2021

                  Key Diagnosis and Treatment Technology of Genetic and Metabolic

                  Not yet recruiting
                  • Diagnosis , Treatment, Genetic and Metabolic Liver Disease
                  • There is no intervention
                  • (no location specified)
                  Oct 27, 2022

                  Biomarker Research in Inherited Movement Disorders

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

                  Whole Exome and Whole Genome Sequencing for Genotyping of

                  Recruiting
                  • Genetic Eye Disease
                    • Bethesda, Maryland
                      National Institutes of Health Clinical Center,
                    Jan 21, 2023

                    COVID-19 and Hereditary Metabolic Diseases

                    Recruiting
                    • Covid19
                    • Metabolism, Inborn Errors
                      • Lille, France
                        Hop Claude Huriez Chu Lille
                      Feb 28, 2022

                      F 18 T807 Tau PET Imaging in Dominantly Inherited Alzheimer's

                      Recruiting
                      • Alzheimer Disease
                      • F 18 T807
                      • Saint Louis, Missouri
                        Washington University School of Medicine
                      Sep 23, 2022

                      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