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Storage disease

Showing 26 - 50 of >10,000
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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

Glycogen Storage Disease Type II Trial in France (Avalglucosidase alfa (GZ402666))

Recruiting
  • Glycogen Storage Disease Type II
  • Avalglucosidase alfa (GZ402666)
  • Brest, France
  • +9 more
Jul 22, 2022

Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency Trial in Israel, United States

Terminated
Has Results
  • Pompe Disease
  • +2 more
  • Myozyme® (alglucosidase alfa)
  • Louisville, Kentucky
  • +4 more
Mar 24, 2022

Lipid Storage Disease Trial in Istanbul

Not yet recruiting
  • Lipid Storage Disease
    • Istanbul, Turkey
      Istanbul University
    Mar 29, 2022

    ERT in Pompe Disease: Elucidation of Molecular Structures

    Not yet recruiting
    • Pompe-Disease
      • Rüsselsheim, Hessen, Germany
        Centre for Analytical Biochemistry
      Jan 30, 2023

      Cognitive and Neurological Pathologies in Pompe Disease

      Recruiting
      • Pompe Disease
        • Durham, North Carolina
          Duke University Medical Center
        Jun 30, 2022

        Glycogen Storage Disease Type II Trial in Worldwide (avalglucosidase alfa)

        Recruiting
        • Glycogen Storage Disease Type II
        • avalglucosidase alfa
        • Leuven, Belgium
        • +7 more
        Jul 13, 2022

        Glycogen Storage Disease Type II Pompe Disease Trial in Worldwide (GZ402666)

        Completed
        • Glycogen Storage Disease Type II Pompe Disease
        • Phoenix, Arizona
        • +16 more
        Dec 14, 2022

        MPS I, MPS II, MPS IVA Trial in San Francisco (Aldurazyme (laronidase))

        Recruiting
        • MPS I
        • +8 more
        • Aldurazyme (laronidase)
        • San Francisco, California
          University of California
        Apr 21, 2022

        Danon Disease Trial in La Jolla, Philadelphia (RP-A501)

        Recruiting
        • Danon Disease
        • RP-A501
        • La Jolla, California
        • +1 more
        Oct 17, 2023

        Orthodontic Appliance Complication Trial in Kuala Lumpur (Hawley retainers in dry storage condition, Hawley retainers in wet

        Recruiting
        • Orthodontic Appliance Complication
        • Hawley retainers in dry storage condition
        • +4 more
        • Kuala Lumpur, Wilayah Persekutuan, Malaysia
          Orthodontic Postgraduate Clinic, Faculty of Dentistry
        May 8, 2023

        Late-onset Pompe Disease Trial in Gainesville, London (Respiratory muscle strength measurements by different techniques.)

        Terminated
        Has Results
        • Late-onset Pompe Disease
        • Respiratory muscle strength measurements by different techniques.
        • Gainesville, Florida
        • +1 more
        Apr 11, 2022

        Pompe's Disease Trial in China (ALGLUCOSIDASE ALFA)

        Recruiting
        • Pompe's Disease
        • ALGLUCOSIDASE ALFA
        • China, China
          Investigational Site
        Oct 26, 2022

        Pompe Telemedicine Developmental Study

        Completed
        • Pompe Disease
        • Glycogen Storage Disease II
          • Durham, North Carolina
            Duke University Medical Center
          Oct 27, 2021

          Avalglucosidase Alfa Pregnancy Study

          Recruiting
          • Pompe Disease
          • Pregnancy
          • avalglucosidase alfa-NGPT (GZ402666) IV
          • avalglucosidase alfa-NGPT (GZ402666)
          • Bridgewater, New Jersey
            Investigational site worldwide
          Feb 15, 2023

          Pompe Disease, Pompe Disease (Late-onset), Pompe's Disease Juvenile Onset Trial in Erlangen (Multispectral Optoacoustic

          Recruiting
          • Pompe Disease
          • +3 more
          • Multispectral Optoacoustic Tomography
          • Erlangen, Bavaria, Germany
            University Hospital Erlangen
          Jun 2, 2022

          Patients With "Late-Onset" Pompe Disease

          Active, not recruiting
          • Pompe Disease
          • +2 more
          • Observational
          • Durham, North Carolina
            Duke University
          Oct 7, 2022

          Pompe Disease (Late-onset) Trial in Durham (Clenbuterol, Placebos)

          Withdrawn
          • Pompe Disease (Late-onset)
          • Durham, North Carolina
            Duke University Medical Center
          Aug 15, 2022

          Biomarker for Glycogen Storage Diseases (BioGlycogen)

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

            Muscular Dystrophies, Congenital Myopathy, Idiopathic Inflammatory Myopathies Trial in Paris (Lower-limb powered dermoskeleton)

            Recruiting
            • Muscular Dystrophies
            • +4 more
            • Lower-limb powered dermoskeleton
            • Paris, France
              Institute of Myology
            Mar 28, 2022

            Glycogen Storage Disease Trial in Conegliano (Low carbohydrate ketogenic diet)

            Completed
            • Glycogen Storage Disease
            • Low carbohydrate ketogenic diet
            • Conegliano, Treviso, Italy
              IRCCS Medea
            Aug 25, 2021

            Iron Deficiency Anemia of Pregnancy, Iron Storage Disease Trial in Jinan (Niferex)

            Recruiting
            • Iron Deficiency Anemia of Pregnancy
            • Iron Storage Disease
            • Jinan, Shandong, China
              Shandong Provincial Qianfoshan Hospital
            Apr 6, 2023

            Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2 Trial in Worldwide (SPK-3006)

            Active, not recruiting
            • Pompe Disease
            • +6 more
            • SPK-3006
            • Phoenix, Arizona
            • +28 more
            Jul 22, 2022

            Late-Onset Pompe Disease, Lysosomal Disease Trial in Durham (IMT therapy using the Pr02 mobile device)

            Recruiting
            • Late-Onset Pompe Disease
            • Lysosomal Disease
            • IMT therapy using the Pr02 mobile device
            • Durham, North Carolina
              Duke University Medical Center
            Jul 21, 2022

            Pompe Disease Infantile-Onset Trial in China (GC301)

            Not yet recruiting
            • Pompe Disease Infantile-Onset
            • GC301
            • Beijing, China
            • +4 more
            Apr 2, 2023