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storage disease

Showing 1 - 25 of >10,000
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Pompe Disease, Muscle Loss, Obesity Trial (Multi-ingredient supplement (PDT-MIS), Placebo (PLA))

Not yet recruiting
  • Pompe Disease
  • +7 more
  • Multi-ingredient supplement (PDT-MIS)
  • Placebo (PLA)
  • (no location specified)
Nov 8, 2023

Biospecimen in Pediatric Patients and Adult Guardians Diagnosed

Recruiting
  • Glycogen Storage Disease Type IB
  • Specimen Donation
  • Woburn, Massachusetts
    Sanguine BioSciences, Inc.
Jun 22, 2023

Glycogen Storage Disease Type IB Trial in Shanghai (Empagliflozin)

Recruiting
  • Glycogen Storage Disease Type IB
  • Shanghai, Shanghai, China
    Xinhua Hospital, Shanghai Jiao Tong University School of Medicin
Jul 17, 2023

Glycogen Storage Disease VI, GLYCOGEN STORAGE DISEASE IXa1, GLYCOGEN STORAGE DISEASE IXa2 Trial in Durham (No intervention)

Recruiting
  • Glycogen Storage Disease VI
  • +4 more
  • No intervention
  • Durham, North Carolina
    Duke University
Jul 27, 2022

Carbon-13 Magnetic Resonance Spectroscopy in Glycogen Storage

Recruiting
  • Glycogen Storage Disease
  • +2 more
  • No intervention
  • Copenhagen, Copenhange, Denmark
    Neuromuscular Research Unit, 8077
Oct 26, 2022

Pompe Disease -Non-invasive Magnetic Resonance Exploratory Pilot

Not yet recruiting
  • Pompe Disease
  • McArdle Disease
  • Siemens Magnetom 7T Plus
  • Vienna, Austria
    Medical University of Vienna
Jul 5, 2023

Hypoglycemia in Glycogen Storage Disease Type III

Recruiting
  • Glycogen Storage Disease Type III
  • No Intervention
  • Philadelphia, Pennsylvania
  • +1 more
Jul 22, 2022

Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease Trial in Gainesville, Decatur, Detroit

Completed
Has Results
  • Pompe Disease
  • +2 more
  • alglucosidase alfa
  • Gainesville, Florida
  • +2 more
Jul 29, 2022

Glycogen Storage Disease Type II, Pompe Disease (Late-onset) Trial in Milan (Inspiratory Muscle Training (IMT), Inspiratory

Recruiting
  • Glycogen Storage Disease Type II
  • Pompe Disease (Late-onset)
  • Inspiratory Muscle Training (IMT)
  • Inspiratory Muscle Training (IMT) + Air Stacking
  • Milan, Italy
    IRCCS S. Maria Nascente - Fondazione Don Carlo Gnocchi
Jul 11, 2023

Glycogen Storage Disease Type IV, Adult Polyglucosan Body Disease Trial in Durham (No intervention)

Recruiting
  • Glycogen Storage Disease Type IV
  • Adult Polyglucosan Body Disease
  • No intervention
  • Durham, North Carolina
    Duke University Medical Center
May 20, 2022

Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV A Trial in San Francisco (There is no intervention)

Recruiting
  • Mucopolysaccharidosis I
  • +7 more
  • There is no intervention
  • San Francisco, California
    University of California San Francisco
Nov 9, 2022

Glycogen Storage Disease Type II Infantile Onset Trial in Gainesville (Cipaglucosidase alfa (ATB200), Miglustat (AT2221))

Recruiting
  • Glycogen Storage Disease Type II Infantile Onset
  • Gainesville, Florida
    University of Florida Clinical Research Center
Jan 18, 2023

Glycogen Storage Disease Type IA Trial in Worldwide (genetic, other, drug)

Recruiting
  • Glycogen Storage Disease Type IA
  • DTX401
  • +3 more
  • Orange, California
  • +16 more
Jan 13, 2023

Adeno-Associated Virus Antibody Study in Subjects OTC

Recruiting
  • Ornithine Transcarbamylase Deficiency
  • +2 more
    • Akron, Ohio
    • +2 more
    Jul 22, 2022

    Glycogen Storage Disease Type IA Trial in Groningen ([6,6-2H2]glucose)

    Completed
    • Glycogen Storage Disease Type IA
    • [6,6-2H2]glucose
    • Groningen, Netherlands
      University of Groningen, UMC Groningen
    Feb 10, 2022

    Glycogen Storage Disease Trial in United States (mRNA-3745)

    Recruiting
    • Glycogen Storage Disease
    • Farmington, Connecticut
    • +4 more
    Jun 10, 2022

    Natural History of Pompe Disease

    Recruiting
    • Glycogen Storage Disease Type II, Adult
      • Garches, Hauts-de-Seine, France
        Hôpital Raymond Poincaré
      Apr 20, 2022

      Pompe Disease (Late-onset) Trial (No Intervention)

      Not yet recruiting
      • Pompe Disease (Late-onset)
      • No Intervention
      • (no location specified)
      Nov 21, 2023

      Glycogen Storage Disease Type III Trial in Worldwide (biological, other, drug)

      Recruiting
      • Glycogen Storage Disease Type III
      • UX053
      • +4 more
      • Orange, California
      • +9 more
      Jul 22, 2022

      GSD1 Trial in Worldwide (DTX401)

      Completed
      Has Results
      • GSD1
      • DTX401
      • steroid regimen
      • Farmington, Connecticut
      • +5 more
      Oct 25, 2022

      Glycogen Storage Disease Type I Trial in Durham (Triheptanoin)

      Completed
      • Glycogen Storage Disease Type I
      • Durham, North Carolina
        Duke University Medical Center
      Dec 14, 2021

      Genetic Studies of Lysosomal Storage Disorders

      Enrolling by invitation
      • Lysosomal Storage Disorders
      • +2 more
        • Bethesda, Maryland
          National Institutes of Health Clinical Center
        Jan 21, 2023

        Glycogen Storage Disease Type II Trial in Worldwide (Alglucosidase alfa GZ419829)

        Recruiting
        • Glycogen Storage Disease Type II
        • Alglucosidase alfa GZ419829
        • Leuven, Belgium
        • +8 more
        Jan 17, 2023

        Inborn Errors of Cholesterol Synthesis and Related Disorders

        Recruiting
        • Lysosomal Storage Disease
        • Cholesterol Metabolism
          • Bethesda, Maryland
            National Institutes of Health Clinical Center
          Dec 28, 2022

          Lysosomal Storage Disease, Metachromatic Leukodystrophy Trial in Milan (OTL-200 Gene Therapy)

          Active, not recruiting
          • Lysosomal Storage Disease
          • Metachromatic Leukodystrophy
          • OTL-200 Gene Therapy
          • Milan, Italy
            Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR
          Nov 4, 2022