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inborn errors of metabolism

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Orphan Drugs for Inherited Metabolic Diseases

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

    Organ Transplant, Cancer, Inborn Errors of Metabolism Trial (A sphygmanometer, a digital thermometer, a pulse oximeter and a

    Not yet recruiting
    • Organ Transplant
    • +5 more
    • A sphygmanometer, a digital thermometer, a pulse oximeter and a peak flow meter will be given to participants to use during their telemedicine consultations with health care providers.
    • (no location specified)
    Feb 8, 2023

    Transition, Inborn Errors of Metabolism Trial in Austria, Switzerland (Behavioral)

    Not yet recruiting
    • Transition
    • Inborn Errors of Metabolism
    • Behavioral
    • Vienna, Austria
    • +7 more
    Nov 9, 2022

    Utilizing Augmented Artificial Intelligence for

    Completed
    • Inborn Errors of Metabolism
    • Plasma amino acid assay
    • Karachi, Sindh, Pakistan
      Aga Khan University Hospital
    Jun 29, 2022

    Primary Immunodeficiencies, Inborn Errors of Metabolism, Haemoglobinopathies Trial in Worldwide (Treosulfan, Busilvex)

    Active, not recruiting
    • Primary Immunodeficiencies
    • +3 more
    • Prague, Czechia
    • +20 more
    Apr 19, 2022

    Intestinal Microbiota in Inborn Errors of Metabolism (IEM)

    Recruiting
    • Inborn Errors of Metabolism
    • +3 more
      • Zürich, Switzerland
        University Children's Hospital
      Apr 8, 2022

      Biomarkers for Inborn Errors of Metabolism

      Completed
      • Inborn Errors of Metabolism
      • Biomarker
        • Tirana, Albania
        • +12 more
        Mar 23, 2022

        Urea Cycle Disorder, Inborn Errors of Metabolism Trial in Philadelphia (UCD Anamix Infant)

        Completed
        • Urea Cycle Disorder
        • Inborn Errors of Metabolism
        • UCD Anamix Infant
        • Philadelphia, Pennsylvania
          Children's hospital of Philadelphia
        Dec 8, 2021

        Leukemia, Inborn Errors of Metabolism, Bone Marrow Failure Syndromes Trial in Philadelphia (Transplant of stem cells with

        Recruiting
        • Leukemia
        • +4 more
        • Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs)
        • Philadelphia, Pennsylvania
          Children's Hospital of Philadelphia
        Nov 2, 2021

        Adult Patients With Inborn Errors of Metabolism in Switzerland

        Completed
        • Inborn Errors of Metabolism
          • Lausanne, Vaud, Switzerland
            Lausanne University Hospitals
          Jul 19, 2021

          Induced Pluripotent Stem Cells for Development of Novel Drug

          Recruiting
          • Inborn Errors of Metabolism
          • Skin biopsy
          • Withdraw of blood
          • Lahore, Pakistan
            Children Hospital and Institute of Child Health
          May 14, 2021

          Multiple Anomalies, Dysmorphic Features, Inborn Errors of Metabolism Trial in Manchester (Face2Gene)

          Completed
          • Multiple Anomalies
          • +2 more
          • Face2Gene
          • Manchester, Greater Manchester, United Kingdom
            Manchester University NHS Foundation Trust
          Jan 13, 2021

          Brain Injuries, Congenital Heart Disease, Newborn Morbidity Trial in São Paulo

          Not yet recruiting
          • Brain Injuries
          • +16 more
            • São Paulo, SP, Brazil
            • +1 more
            Nov 11, 2020

            Measurements in Inborn Errors of Metabolism

            Completed
            • Inborn Errors of Metabolism
            • +7 more
            • Survey
            • Zurich, Switzerland
              University Children's Hospital Zurich
            Nov 3, 2020

            Hurler Syndrome, Inherited Metabolic Disorder, Lysosomal Storage Disorder Trial in Los Angeles, Palo Alto (rivogenlecleucel,

            No longer available
            • Hurler Syndrome
            • +4 more
            • Los Angeles, California
            • +1 more
            Oct 1, 2020

            PKU, Phenylketonurias, Inborn Errors of Metabolism Trial in Birmingham, Bristol (GMPOWER)

            Completed
            • PKU
            • +3 more
            • GMPOWER
            • Birmingham, United Kingdom
            • +1 more
            Mar 20, 2020

            PKU, Phenylketonurias, Inborn Errors of Metabolism Trial in Birmingham (Motion)

            Completed
            • PKU
            • +3 more
            • Motion
            • Birmingham, United Kingdom
              Birmingham Children's Hospital
            Mar 12, 2020

            Inborn Errors of Metabolism Trial in Washington (N-carbamylglutamate)

            Unknown status
            • Inborn Errors of Metabolism
            • Washington, District of Columbia
              Childrens Research Institute
            Feb 13, 2020

            Mitochondrial Diseases, Metabolic Disease, Inborn Errors of Metabolism Trial (EE-TP)

            Not yet recruiting
            • Mitochondrial Diseases
            • +2 more
            • EE-TP
            • (no location specified)
            Nov 4, 2019

            Leukemia, Lymphoma, Bone Marrow Failure Trial in Valhalla (Full Intensity with TBI, Full Intensity, Reduced Intensity)

            Completed
            • Leukemia
            • +7 more
            • Full Intensity with TBI
            • +3 more
            • Valhalla, New York
              New York Medical College
            Aug 20, 2018

            Phenylketonuria, Inborn Errors of Metabolism Trial in Glasgow, Burringham, Bradford (PKU Explore)

            Completed
            • Phenylketonuria
            • Inborn Errors of Metabolism
            • PKU Explore
            • Glasgow, Lanarkshire, United Kingdom
            • +2 more
            Apr 4, 2018

            Albinism, Inborn Errors of Metabolism, Oculocutaneous Albinism Trial run by the (Pirfenidone, Placebo)

            Completed
            Has Results
            • Albinism
            • +4 more
            • Bethesda, Maryland
              National Institutes of Health Clinical Center, 9000 Rockville Pi
            Sep 14, 2017

            Phenylketonuria, Inborn Errors of Metabolism Trial in Glasgow, Birmingham, Bradford (PKU Start)

            Completed
            • Phenylketonuria
            • Inborn Errors of Metabolism
            • PKU Start
            • Glasgow, Lanarkshire, United Kingdom
            • +2 more
            Sep 26, 2017

            Lysosomal Storage Diseases, Inborn Errors of Metabolism Trial in Durham (ALD-601)

            Withdrawn
            • Lysosomal Storage Diseases
            • Inborn Errors of Metabolism
            • ALD-601
            • Durham, North Carolina
            • +1 more
            Jun 27, 2017

            MDS, Anemia, Aplastic, Inborn Errors of Metabolism Trial in Durham (ALDHbr Umbilical Cord Blood Cells)

            Completed
            • MDS
            • +4 more
            • ALDHbr Umbilical Cord Blood Cells
            • Durham, North Carolina
              Duke University Medical Center
            Jun 27, 2017