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Refsum Disease, Infantile

Showing 1 - 25 of >10,000
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Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome Trial in Minneapolis (Stem Cell Transplantation, IMD

Recruiting
  • Mucopolysaccharidosis Disorders
  • +28 more
  • Stem Cell Transplantation
  • +7 more
  • Minneapolis, Minnesota
    Masonic Cancer Center, University of Minnesota
Nov 1, 2022

Infantile Refsum's Disease, Zellweger Syndrome, Adrenoleukodystrophy Trial in Cincinnati (Cholic Acids)

Completed
Has Results
  • Infantile Refsum's Disease
  • +4 more
  • Cholic Acids
  • Cincinnati, Ohio
    Cincinnati Children's Hospital Medical Center
Sep 28, 2020

Pompe Disease Infantile-Onset Trial in China (GC301)

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

Krabbe Disease Trial in Ann Arbor (FBX-101)

Recruiting
  • Krabbe Disease
  • FBX-101
  • Ann Arbor, Michigan
    University of Michigan Hospitals - Michigan Medicine
Feb 13, 2023

Infantile-onset Pompe Disease Trial in Beijing (Genetic: GC301)

Recruiting
  • Infantile-onset Pompe Disease
  • Genetic: GC301
  • Beijing, Beijing, China
    Bayi Children's Hospital, Seventh Medical Center, PLA general ho
Sep 30, 2022

Patients With Peroxisome Biogenesis Disorders (PBD)

Recruiting
  • Peroxisome Biogenesis Disorder
  • +10 more
    • Montreal, Quebec, Canada
      Research Institute of the McGill University Health Center
    Jul 4, 2022

    Infantile GM2 Gangliosidosis (Disorder) Trial in Kingston (TSHA-101)

    Active, not recruiting
    • Infantile GM2 Gangliosidosis (Disorder)
    • TSHA-101
    • Kingston, Ontario, Canada
      Queen's University/Kingston Health Sciences Centre
    Sep 12, 2022

    GM2 Gangliosidosis, Supportive Care Trial in Kashan, Mashhad, Tehran (Miglustat)

    Recruiting
    • GM2 Gangliosidosis
    • Supportive Care
    • Kashan, Isfahan, Iran, Islamic Republic of
    • +2 more
    Oct 23, 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

    Variant Late-Infantile Neuronal Ceroid Lipofuscinosis Trial in Columbus (AT-GTX-501)

    Completed
    • Variant Late-Infantile Neuronal Ceroid Lipofuscinosis
    • AT-GTX-501
    • Columbus, Ohio
      Nationwide Children's Hosptial
    Jan 21, 2022

    Late Infantile Variant CLN5 And CLN7 Disease

    Recruiting
    • Ceroid Lipofuscinosis, Neuronal 5
    • Ceroid Lipofuscinosis, Neuronal 7
      • Rochester, New York
        University of Rochester Medical Center
      Mar 1, 2022

      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

      Intestinal Transglutaminase Antibodies in Celiac Disease

      Completed
      • Celiac Disease
      • Evaluation of diagnostic accuracy of anti-ttg-m
      • Trieste, Italy
        IRCCS materno infantile Burlo Garofolo
      Apr 11, 2023

      Models of Enteric Disease

      Recruiting
      • Diarrhea, Infantile
      • +4 more
        • Nairobi, Kenya
          African Population Health Research Center
        Aug 2, 2022

        Glycogen Storage Disease Type II-Pompe's Disease Trial in Worldwide (Avalglucosidase alfa (GZ402666), Alglucosidase alfa

        Active, not recruiting
        Has Results
        • Glycogen Storage Disease Type II-Pompe's Disease
        • Avalglucosidase alfa (GZ402666)
        • Alglucosidase alfa (GZ419829)
        • Valhalla, New York
        • +9 more
        May 3, 2022

        A Natural History Study of Charcot-Marie-Tooth 4J (CMT4J)

        Terminated
        • Charcot-Marie-Tooth Disease
        • Hereditary Motor and Sensory Neuropathy IV
          • Iowa City, Iowa
          • +1 more
          Mar 16, 2022

          Leukodystrophy, Globoid Cell Trial in Worldwide (PBKR03)

          Recruiting
          • Leukodystrophy, Globoid Cell
          • PBKR03
          • Chicago, Illinois
          • +8 more
          Aug 17, 2022

          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

          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

          Interstitial Lung and Liver Disease, Infantile Liver Failure Syndrome 1, Neurologic, Endocrine and Pancreatic Disease,

          Not yet recruiting
          • Interstitial Lung and Liver Disease
          • +3 more
          • Procedure/Surgery
          • Paris, France
            Hôpital Necker-Enfants Malades
          Aug 22, 2022

          Death, Death, Infant, Diarrhea Trial in Kisumu, Siaya (Coupons for safe water program: Treatment, Coupons for safe water

          Recruiting
          • Death
          • +5 more
          • Coupons for safe water program: Treatment
          • Coupons for safe water program: Control
          • Kisumu, Kenya
          • +1 more
          Mar 10, 2023

          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

          Pompe Disease Infantile-Onset Trial in Italy, Taiwan, United States (ATB200, AT2221)

          Available
          • Pompe Disease Infantile-Onset
          • Oakland, California
          • +3 more
          Jul 18, 2022

          Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence Trial in Sioux Falls, Sydney

          Recruiting
          • Rare Disorders
          • +309 more
            • Sioux Falls, South Dakota
            • +1 more
            Jun 20, 2022

            Autism Spectrum Disorder (ASD) in Toddlers

            Recruiting
            • Autism Spectrum Disorder
            • +4 more
            • Treatment Monitoring by Expert Clinician During Behavioral Intervention (ABA or similar)
            • Treatment Monitoring by the EarliPoint Assessment For Autism Spectrum Disorder
            • Phoenix, Arizona
            • +1 more
            Jan 27, 2023