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Inborn errors of metabolism

Showing 26 - 50 of 6,907
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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

Methylmalonic Acidemia (MMA) Trial run by the Selecta Biosciences, Inc. (SEL-302)

Recruiting
  • Methylmalonic Acidemia (MMA)
  • Bethesda, Maryland
    National Human Genome Research Institute, National Institutes of
Mar 9, 2023

Patients Who Received hLB-001 Gene Therapy

Recruiting
  • Methylmalonic Acidemia
  • Nashville, Tennessee
  • +1 more
Aug 17, 2022

Cardiac Manifestation and Inherited Metabolic Diseases

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

    Methylmalonic Acidemia, Propionic Acidemia Trial in San Francisco, Remote (Registry)

    Recruiting
    • Methylmalonic Acidemia
    • Propionic Acidemia
    • Registry
    • San Francisco, California
    • +2 more
    Jun 27, 2022

    Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell

    Completed
    • Hematologic Malignancies
    • +7 more
    • San Francisco, California
      University of California, San Francisco
    Sep 27, 2021

    Fludarabine Drug Exposure in Pediatric Bone Marrow

    Completed
    • Hematologic Malignancies
    • +7 more
    • San Francisco, California
      University of California, San Francisco
    Sep 27, 2021

    Clofarabine and Fludarabine Drug Exposure in Pediatric Bone

    Completed
    • Hematologic Malignancies
    • +7 more
    • San Francisco, California
      University of California, San Francisco
    Sep 27, 2021

    Methylmalonic Acidemia Trial in United States (hLB-001)

    Recruiting
    • Methylmalonic Acidemia
    • hLB-001
    • Atlanta, Georgia
    • +3 more
    Aug 16, 2022

    Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies Trial in Durham (CliniMACS CD34 Reagent

    Available
    • Hematologic Malignancies
    • +2 more
    • CliniMACS CD34 Reagent System
    • Durham, North Carolina
      Duke University Medical Center
    Nov 12, 2021

    Congenital Sucrase-Isomaltase Deficiency, CSID, Sucrase Isomaltase Deficiency Trial in Buffalo, Oklahoma City (Sucraid

    Recruiting
    • Congenital Sucrase-Isomaltase Deficiency
    • +2 more
    • Sucraid (sacrosidase) Oral Solution 8500 IU/mL
    • Buffalo, New York
    • +1 more
    Aug 16, 2022

    Pyruvate Kinase Deficiency, Pyruvate Kinase Deficiency Anemia, Anemia Trial in Worldwide

    Recruiting
    • Pyruvate Kinase Deficiency
    • +3 more
      • Boston, Massachusetts
      • +4 more
      Aug 5, 2022

      Carbaglu® for Treatment of MMA and PA in Adults and Pediatrics

      Recruiting
      • Hyperammonemia
      • +2 more
      • Carglumic Acid
      • Washington, District of Columbia
      • +1 more
      Jun 30, 2022

      Irritable Bowel Syndrome, Sucrose Intolerance Due to Sucrase-Isomaltase Deficiency, Carbohydrate; Malabsorption Trial

      Not yet recruiting
      • Irritable Bowel Syndrome
      • +4 more
      • Low-FODMAP diet
      • Starch- and Sucrose Reduced Diet (SSRD)
      • (no location specified)
      Jan 18, 2022

      Measurements in Inborn Errors of Metabolism

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

      Non Infectious Presentation of Inborn Errors Of Immunity

      Not yet recruiting
      • Inborn Errors of Immunity
        • Sohag, Egypt
          Sohag University Hospital
        May 21, 2023

        Exposure to Pollution, Glucocorticoid Resistance Trial in Vancouver (Placebo, Budesonide, Filtered Air)

        Recruiting
        • Exposure to Pollution
        • Glucocorticoid Resistance
        • Placebo
        • +3 more
        • Vancouver, British Columbia, Canada
          University of British Columbia
        Oct 29, 2021

        Molybdenum Cofactor Deficiency, Type A Trial in Worldwide (ORGN001 (formerly ALXN1101))

        Completed
        • Molybdenum Cofactor Deficiency, Type A
        • ORGN001 (formerly ALXN1101)
        • Milwaukee, Wisconsin
        • +5 more
        Oct 13, 2022

        Prospective Research Rare Kidney Stones (ProRKS)

        Recruiting
        • Hyperoxaluria
        • +4 more
          • Birmingham, Alabama
          • +10 more
          Nov 10, 2022

          Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia Trial in Barcelona, Madrid (Mitapivat, Mitapivat-matching )

          Recruiting
          • Pediatric Pyruvate Kinase Deficiency
          • Pediatric Hemolytic Anemia
          • Barcelona, Spain
          • +1 more
          Aug 8, 2022

          GLUT1 Deficiency Syndrome, Glucose Transporter Type 1 Deficiency Syndrome, Glucose Transporter type1 (GLUT-1) Deficiency Trial

          Recruiting
          • GLUT1 Deficiency Syndrome
          • +3 more
          • No intervention
          • Dallas, Texas
            UT Southwestern Medical Center
          Feb 1, 2022

          Biomarker for Hurler Disease (BioHurler)

          Active, not recruiting
          • Mucopolysaccharidosis Type I
          • +2 more
            • Cairo, Egypt
            • +4 more
            May 12, 2021

            Biomarker for Glycogen Storage Diseases (BioGlycogen)

            Active, not recruiting
            • Fructose Metabolism, Inborn Errors
            • +9 more
              • Rostock, Germany
              • +3 more
              May 12, 2021

              Pearson Syndrome Natural History Study

              Recruiting
              • Pearson Syndrome
              • Single Large Scale Mitochondrial DNA Deletion Syndromes (SLSMDS)
                • Cleveland, Ohio
                • +1 more
                Aug 30, 2021