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Methylmalonic acidemia with homocystinuria,

Showing 1 - 25 of 72
STUDY
CONDITIONS
INTERVENTIONS
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Propionic/Methylmalonic Acidemias, Maple Syrup Urine Disease, Citrullinemia Trial in Aktobe (Obtaining Dry Blood Spots From

Recruiting
  • Propionic/Methylmalonic Acidemias
  • +28 more
  • Obtaining Dry Blood Spots From Healthy Newborns (Aged 1-7 Days)
  • +5 more
  • Aktobe, Aktobe Region, Kazakhstan
    Regional perinatal center of Aktobe region
Jun 8, 2023

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

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

Baby Detect : Genomic Newborn Screening

Recruiting
  • Congenital Adrenal Hyperplasia
  • +126 more
    • Liege, Wallonia, Belgium
      CRMN, Hôpital La Citadelle
    Jan 24, 2023

    Healthy Volunteers, Propionic Acidemia, Methylmalonic Acidemia Trial in Topeka, Pittsburgh, Austin (BBP-671, Placebo)

    Recruiting
    • Healthy Volunteers
    • +3 more
    • Topeka, Indiana
    • +2 more
    Apr 8, 2022

    Methylmalonic Acidemia Trial in United States (hLB-001)

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

    Methylmalonic Acidemia, Propionic Acidemia Trial in United States (HST5040, Placebo)

    Recruiting
    • Methylmalonic Acidemia
    • Propionic Acidemia
    • San Diego, California
    • +10 more
    Sep 6, 2022

    A Non-Interventional Post-Authorization Study of Carbaglu for

    Enrolling by invitation
    • Hyperammonemia
    • +2 more
    • carglumic acid
    • Indianapolis, Indiana
      Riley Children's Hospital
    Apr 24, 2023

    Methylmalonic Acidemia Trial in Birmingham (mRNA-3705)

    Recruiting
    • Methylmalonic Acidemia
    • Toronto, Ontario, Canada
    • +1 more
    Dec 20, 2022

    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

    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

    Patients Who Received hLB-001 Gene Therapy

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

      Methylmalonic Acidemia (MMA), Metabolism, Inborn Errors Trial (mRNA-3704)

      Withdrawn
      • Methylmalonic Acidemia (MMA)
      • Metabolism, Inborn Errors
      • mRNA-3704
      • (no location specified)
      Nov 10, 2020

      Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA) Trial in Taipei (Carglumic Acid (Carbaglu®))

      Unknown status
      • Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA)
      • Carglumic Acid (Carbaglu®)
      • Taipei, Taiwan
        National Taiwan University Hospital
      Feb 25, 2020

      Amino Acid Metabolism, Inborn Errors Trial in Worldwide (Ataluren)

      Terminated
      Has Results
      • Amino Acid Metabolism, Inborn Errors
      • Antwerp, Belgium
      • +8 more
      Jun 22, 2020

      "The MaP Study": Mapping the Patient Journey in MMA and PA

      Completed
      • Methylmalonic Acidemia
      • Propionic Acidemia
        • Stanford, California
        • +16 more
        Jul 29, 2021

        Organic Acidemia CARBAGLU®: A Mixed Methods Approach

        Unknown status
        • Methylmalonic Acidemia
        • Propionic Acidemia
        • Carglumic Acid
        • Bordeaux, France
        • +20 more
        Jan 8, 2020

        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

        Propionic Acidemia, Methylmalonic Acidemia Trial in Riyadh (Carglumic Acid)

        Completed
        • Propionic Acidemia
        • Methylmalonic Acidemia
        • Carglumic Acid
        • Riyadh, Saudi Arabia
        • +1 more
        Apr 24, 2019

        Systemic Biomarkers of Brain Injury From Hyperammonemia

        Recruiting
        • Urea Cycle Disorder
        • +5 more
          • Washington, District of Columbia
            Children's National Research Institute
          Oct 25, 2021

          Homocystinuria Trial in United States (Pegtibatinase, Placebo)

          Recruiting
          • Homocystinuria
          • Aurora, Colorado
          • +6 more
          Jul 11, 2022

          Characterize Participants With Propionic Acidemia

          Not yet recruiting
          • Propionic Acidemia
            • (no location specified)
            Mar 3, 2023

            Homocystinuria Due to Cystathionine Beta-Synthase Deficiency Trial in Australia, United Kingdom, United States (Pegtarviliase)

            Recruiting
            • Homocystinuria Due to Cystathionine Beta-Synthase Deficiency
            • Dallas, Texas
            • +7 more
            Aug 18, 2022

            Fetal Acidemia Trial in Beijing (Optiflow high-flow nasal cannula system)

            Not yet recruiting
            • Fetal Acidemia
            • Optiflow high-flow nasal cannula system
            • Beijing, Beijing, China
              Beijing tongren Hospital, Capital Medical University
            Jun 25, 2023

            Homocystinuria Trial in Paris (Betaine)

            Completed
            • Homocystinuria
            • Paris, France
              Assistance Publique - Hôpitaux de Paris
            Feb 1, 2023