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Mucopolysaccharidosis IV

Showing 1 - 25 of 9,074
STUDY
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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

MPS (RaDiCo Cohort) (RaDiCo-MPS)

Recruiting
  • Mucopolysaccharidosis I
  • +7 more
    • Angers, France
    • +22 more
    Sep 12, 2023

    Osteochondrodysplasia Trial in Parkville (SAR442501)

    Recruiting
    • Osteochondrodysplasia
    • Parkville, Victoria, Australia
      Investigational Site Number: 0360001
    Oct 10, 2023

    Natural History of Atypical Morquio A Disease

    Active, not recruiting
    • Mucopolysaccharidosis IV A
    • Elosulfase Alfa 1 MG/ML Intravenous Solution [VIMIZIM]
    • Bordeaux, Aquitaine, France
      Rheumatology department - Bordeaux University Hospital
    Feb 15, 2022

    MPS IVA, MPS VI Trial in Oakland (Vosoritide Injection [Voxzogo])

    Not yet recruiting
    • MPS IVA
    • MPS VI
    • Vosoritide Injection [Voxzogo]
    • Oakland, California
      UCSF Benioff Children's Hospital Oakland
    May 4, 2023

    Osteochondrodysplasia Trial in Newark (SAR442501, Placebo)

    Completed
    • Osteochondrodysplasia
    • Newark, New Jersey
      Investigational site
    Apr 26, 2023

    Non-invasive Functional Assessment and Pathogenesis of Morquio A

    Recruiting
    • Mucopolysaccharidosis IV Type A
    • +2 more
    • Imaging, gait analysis, growth, joint test, hearing test, questionnaire, etc.
    • Wilmington, Delaware
      Nemours Children's Health, Delaware Valley
    Mar 9, 2022

    ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn

    Enrolling by invitation
    • Acid Sphingomyelinase Deficiency
    • +13 more
    • Confirmatory Testing
    • Bronx, New York
    • +9 more
    May 9, 2022

    Baby Detect : Genomic Newborn Screening

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

      Hunter Syndrome Trial in India (Elaprase)

      Recruiting
      • Hunter Syndrome
      • Elaprase
      • Thiruvananthapuram, Kerala, India
      • +4 more
      May 5, 2022

      Mucopolysaccharidosis IV A, Morquio A Syndrome, MPS IVA Trial in Worldwide (BMN 110 - Weekly, BMN 110 - Every Other Week)

      Completed
      Has Results
      • Mucopolysaccharidosis IV A
      • +2 more
      • BMN 110 - Weekly
      • BMN 110 - Every Other Week
      • Phoenix, Arizona
      • +42 more
      Jul 19, 2021

      Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidoses Trial in Porto Alegre (Losartan, Placebo)

      Recruiting
      • Mucopolysaccharidosis IV A
      • +8 more
      • Porto Alegre, Rio Grande Do Sul, Brazil
        Hospital de Clinicas de Porto Alegre
      Oct 2, 2020

      Human Cellular Disease Models for Morquio Disease

      Completed
      • Morquio Disease
        • Lahore, Pakistan
          Childrens Hospital and Institute of Child Health, Ferozepur Road
        Apr 8, 2021

        Pregnancy With Morquio Syndrome - What Are Patients'

        Completed
        • Morquio Disease
        • MPS - Mucopolysaccharidosis
        • Interview
        • Decatur, Georgia
          Emory University
        Jan 15, 2021

        The Natural History of Metachromatic Leukodystrophy

        Recruiting
        • MLD
        • +26 more
        • Palliative Care
        • Hematopoetic Stem Cell Transplantation
        • Pittsburgh, Pennsylvania
          The Program for the Study of Neurodevelopment in Rare Disorders,
        Nov 2, 2021

        Biomarker for Morquio Disease (BioMorquio)

        Active, not recruiting
        • Morquio Syndrome
        • +3 more
          • Cairo, Egypt
          • +4 more
          May 12, 2021

          Mucopolysaccharidosis IV Type A, Morquio A Syndrome, MPS IVA Trial in Worldwide (Vimizim® (elosulfase alfa))

          Active, not recruiting
          • Mucopolysaccharidosis IV Type A
          • +2 more
          • Vimizim® (elosulfase alfa)
          • Phoenix, Arizona
          • +78 more
          Dec 23, 2022

          Gangliosidoses, GM1 Gangliosidosis, Morquio B Disease Trial in Hochheim

          Recruiting
          • Gangliosidoses
          • +8 more
            • Hochheim, HE, Germany
              SphinCS Lyso gemeinnützige UG (haftungsbeschränkt)
            Nov 5, 2020

            Before and After Hematopoietic Cell Transplantation in

            Enrolling by invitation
            • Hematopoietic Cell Transplantation
            • Mucopolysaccharidosis Type I
            • Laronidase therapy and a stem cell transplant
            • Minneapolis, Minnesota
              University of Minnesota Masonic Cancer Center
            Nov 22, 2022

            Mucopolysaccharidosis III-A Trial in Hamburg (JR-441)

            Recruiting
            • Mucopolysaccharidosis III-A
            • Hamburg, Germany
              Universitätsklinikum Hamburg-Eppendorf
            Oct 18, 2023

            Mucopolysaccharidosis II Trial in Japan (JR-141)

            Completed
            • Mucopolysaccharidosis II
            • Gifu, Japan
            • +7 more
            Nov 10, 2022

            Brain Structure and Function in MPS Disorders

            Completed
            • Mucopolysaccharidosis Type I
            • +4 more
              • Oakland, California
              • +3 more
              Sep 4, 2019

              Mucopolysaccharidosis II Trial in Porto Alegre, São Paulo (JR-141)

              Completed
              • Mucopolysaccharidosis II
              • Porto Alegre, Brazil
              • +1 more
              Nov 10, 2022

              Gait Analysis in MPS IVA

              Completed
              • MPS IVA
              • Morquio Syndrome
                • Portland, Oregon
                  Oregon Health and Science University
                Oct 15, 2019

                Mucopolysaccharidosis I Trial in Brazil, Japan, United States (JR-171)

                Completed
                • Mucopolysaccharidosis I
                • Oakland, California
                • +5 more
                Aug 16, 2022