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Methylmalonic Acidemia

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

Recruiting
  • Methylmalonic Acidemia
  • Propionic Acidemia
  • Registry
  • San Francisco, California
  • +2 more
Jun 27, 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 United States (hLB-001)

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

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

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

Patients Who Received hLB-001 Gene Therapy

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

Methylmalonic Acidemia Trial in Birmingham (mRNA-3705)

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Withdrawn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

Intestinal Microbiota in Inborn Errors of Metabolism (IEM)

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

    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

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

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

      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

      Baby Detect : Genomic Newborn Screening

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

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

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

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

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

        Organic Acidemia CARBAGLU®: A Mixed Methods Approach

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

        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

          Seeks to Understand Clinical, Genomic, Pharmacological,

          Not yet recruiting
          • Purine-Pyrimidine Metabolism
          • Metabolic Disease
            • Bethesda, Maryland
              National Institutes of Health Clinical Center
            Oct 26, 2023

            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

            Characterize Participants With Propionic Acidemia

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

              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

              Metabolic Acidosis, Acute Kidney Injury Trial in Montpellier (Sodium bicarbonate infusion)

              Recruiting
              • Metabolic Acidosis
              • Acute Kidney Injury
              • Sodium bicarbonate infusion
              • Montpellier, France
                Service de médecine intensive et réanimation
              Feb 11, 2022

              Organic Acidemia, Cognitive Orientation Trial in Ankara (The Cognitive Orientation to Daily Occupational Performance (CO-OP)

              Completed
              • Organic Acidemia
              • Cognitive Orientation
              • The Cognitive Orientation to Daily Occupational Performance (CO-OP) Approach
              • Home Program
              • Ankara, Altindag, Turkey
                Hacettepe University
              Oct 13, 2021