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Inherited metabolic diseases

Showing 1 - 25 of >10,000
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Orphan Drugs for Inherited Metabolic Diseases

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

    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

    Inherited Diseases, Caregiving, and Social Networks

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

      Cardiac Manifestation and Inherited Metabolic Diseases

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

        Studies of Inherited Diseases of Metabolism

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

          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

          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

            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

            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

            Global Patient Registry of Inherited Retinal Diseases

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

            Pattern of Metabolic Causes of Neonatal Hypoglycemia

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

              Families With Inherited Eye Disease

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

                Repository for Inherited Eye Diseases

                Active, not recruiting
                • Retinitis Pigmentosa
                • Inherited Ophthalmic Diseases
                  • Bethesda, Maryland
                    National Institutes of Health Clinical Center, 9000 Rockville Pi
                  Aug 24, 2022

                  Study of Inherited Neurological Disorders

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

                    COVID-19 and Hereditary Metabolic Diseases

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

                      Incidence of Metabolic Disorders in Neonatal Convulsions

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

                      Maternal Endocrine System and Metabolic Diseases and Offspring

                      Not yet recruiting
                      • Thyroid Dysfunction
                      • Diabetes Mellitus
                        • (no location specified)
                        Jul 17, 2023

                        Inherited Reproductive Disorders

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