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MPS IV

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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

MPS (RaDiCo Cohort) (RaDiCo-MPS)

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

    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

    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

    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

    Pregnancy With Morquio Syndrome - What Are Patients'

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

    MPS-IH (Hurler Syndrome) Trial in Worldwide (Experimental: OTL-203, Active Comparator: Allo-HSCT)

    Not yet recruiting
    • MPS-IH (Hurler Syndrome)
    • Experimental: OTL-203
    • Active Comparator: Allo-HSCT
    • Oakland, California
    • +6 more
    Nov 27, 2023

    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 Type I (MPS I), Hurler Syndrome, Hurler-Scheie Syndrome Trial in Orange, Philadelphia, Porto Alegre

    Active, not recruiting
    • Mucopolysaccharidosis Type I (MPS I)
    • +2 more
    • RGX-111
    • Orange, California
    • +3 more
    Jan 9, 2023

    Gait Analysis in MPS IVA

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

      Mucopolysaccharidosis I Trial in Wuhan (Laronidase)

      Recruiting
      • Mucopolysaccharidosis I
      • Wuhan, China
        Investigational Site Number :1560001
      Jan 15, 2023

      Mucopolysaccharidosis (MPS), Hunter Syndrome Trial (ELAPRASE, Rituximab, Methotrexate)

      Not yet recruiting
      • Mucopolysaccharidosis (MPS)
      • Hunter Syndrome
      • (no location specified)
      Aug 8, 2022

      Mucopolysaccharidosis II Trial in Japan (JR-141)

      Completed
      • Mucopolysaccharidosis II
      • Fukui, Japan
      • +18 more
      Nov 10, 2022

      MPS I Trial in Oakland (SB-318)

      Terminated
      Has Results
      • MPS I
      • SB-318
      • Oakland, California
        UCSF Benioff Children's Hospital Oakland
      Jan 6, 2023

      Mucopolysaccharidosis Type II, Metabolic Diseases Trial in Russian Federation (GNR-055 1.0-2.0-3.0 mg/kg, GNR-055 2.0 mg/kg,

      Recruiting
      • Mucopolysaccharidosis Type II
      • Metabolic Diseases
      • GNR-055 1.0-2.0-3.0 mg/kg
      • +2 more
      • Ekaterinburg, Russian Federation
      • +4 more
      Jan 13, 2022

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

      Active, not recruiting
      • Mucopolysaccharidosis I
      • Oakland, California
      • +4 more
      Aug 16, 2022

      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

      Brain Structure and Function in MPS Disorders

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

        Mucopolysaccharidosis Type 3 B Trial in Worldwide (rAAV9.CMV.hNAGLU)

        Terminated
        • Mucopolysaccharidosis Type 3 B
        • rAAV9.CMV.hNAGLU
        • Columbus, Ohio
        • +3 more
        Apr 29, 2022

        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

        Kidney Transplant Rejection Trial in Montréal (AT-1501, Tacrolimus)

        Enrolling by invitation
        • Kidney Transplant Rejection
        • Montréal, Quebec, Canada
          McGill University Health Care Centre
        Nov 6, 2023

        Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome Trial in Rome (Aldurazyme (laronidase))

        Recruiting
        • Mucopolysaccharidosis I
        • +3 more
        • Aldurazyme (laronidase)
        • Rome, Italy
          Dipartimento di Scienze Pediatriche Medico - Chirurgiche e Neuro
        Mar 28, 2022

        Mucopolysaccharidosis II, MPS II Trial in United States (SB-913)

        Terminated
        Has Results
        • Mucopolysaccharidosis II
        • MPS II
        • SB-913
        • Oakland, California
        • +4 more
        Oct 21, 2022

        Healthy Aging Trial in Exeter (Controlled diet and resistance exercise)

        Recruiting
        • Healthy Aging
        • Controlled diet and resistance exercise
        • Exeter, Devond, United Kingdom
          University of Exeter
        Aug 2, 2023