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MPS IV Type A

Showing 1 - 25 of >10,000
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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

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

MPS (RaDiCo Cohort) (RaDiCo-MPS)

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

    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-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 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

    Mucopolysaccharidosis II Trial in Cincinnati (DNL310)

    Enrolling by invitation
    • Mucopolysaccharidosis II
    • Cincinnati, Ohio
      Cincinnati Children's Hospital Medical Center
    Oct 10, 2023

    Mucopolysaccharidosis Type IIIB, MPS III B Trial in Worldwide (AX 250)

    Active, not recruiting
    • Mucopolysaccharidosis Type IIIB
    • MPS III B
    • AX 250
    • Oakland, California
    • +7 more
    Jan 16, 2023

    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

    Mucopolysaccharidosis Type II (MPS II) Trial in Oakland, Montréal (RGX-121)

    Recruiting
    • Mucopolysaccharidosis Type II (MPS II)
    • RGX-121
    • Oakland, California
    • +1 more
    Oct 6, 2022

    Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS Trial in Minneapolis (Autologous Plasmablasts (B cells))

    Not yet recruiting
    • Mucopolysaccharidosis IH/S
    • Mucopolysaccharidosis IS
    • Autologous Plasmablasts (B cells)
    • Minneapolis, Minnesota
      University of Minnesota
    Jan 20, 2023

    Mucopolysaccharidosis Type II (MPS II) Trial in Brazil, United States (RGX-121)

    Recruiting
    • Mucopolysaccharidosis Type II (MPS II)
    • RGX-121
    • Oakland, California
    • +4 more
    Dec 22, 2022

    Mucopolysaccharidosis II Trial in Salt Lake City, Fairfax (Genetic HMI-203)

    Recruiting
    • Mucopolysaccharidosis II
    • Genetic HMI-203
    • Oakland, California
    • +5 more
    Jan 26, 2023

    Participants With Sanfilippo Syndrome Type IIIC

    Not yet recruiting
    • Sanfilippo Syndrome Type C
      • Bron, France
        Hospices Civils De Lyon
      Apr 20, 2023

      MPS III B Trial in Worldwide (AX 250)

      Not yet recruiting
      • MPS III B
      • AX 250
      • Oakland, California
      • +4 more
      Aug 8, 2022

      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

      Mucolipidosis Type IV Natural History Study

      Recruiting
      • Mucolipidosis Type IV
        • Boston, Massachusetts
          Massachusetts General Hospital
        Mar 13, 2023

        Mucopolysaccharidosis Type II Observational

        Withdrawn
        • Mucopolysaccharidosis II
        • Observational
        • Oakland, California
        • +2 more
        Oct 6, 2022

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

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

        Sanfilippo Syndrome Type A (MPS IIIA) Trial in Oakland, Chapel Hill, Ankara (SOBI003)

        Completed
        Has Results
        • Sanfilippo Syndrome Type A (MPS IIIA)
        • Oakland, California
        • +2 more
        Feb 2, 2022

        Myozyme® and of Aldurazyme® in Male and Female Participants of

        Recruiting
        • Pompe Disease
        • Mucopolysaccharidosis Type I (MPS I)
          • Italy, Italy
            Investigational site Italy
          May 3, 2022

          Mucopolysaccharidosis Type IH (MPS IH, Hurler Syndrome), Mucopolysaccharidosis Type IH, MPS IH, Hurler Syndrome Trial

          Withdrawn
          • Mucopolysaccharidosis Type IH (MPS IH, Hurler Syndrome)
          • +2 more
          • Autologous Plasmablasts
          • (no location specified)
          Oct 5, 2022

          Dental Implant Trial (Type I Dental implant placement., Type II Dental implant placement., Type III/IV dental implant

          Not yet recruiting
          • Dental Implant
          • Type I Dental implant placement.
          • +2 more
          • (no location specified)
          Aug 4, 2023

          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

          Type 1 Diabetes, Type 2 Diabetes, Hyperglycemia Trial in Winston-Salem (Libre 2.0 CGM in patients taking subcutaneous insulin

          Not yet recruiting
          • Type 1 Diabetes Mellitus
          • +2 more
          • Libre 2.0 CGM in patients taking subcutaneous insulin with concomitant high dose glucocorticoids
          • Winston-Salem, North Carolina
            WakeForest University Health Sciences
          May 11, 2023