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MPS I

Showing 1 - 25 of >10,000
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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

Examinations for Asymptomatic MPS I Infants in Taiwan

Recruiting
  • MPS I
  • MPS I
  • Taipei, Taiwan
    Mackay Memorial Hospital
Mar 2, 2022

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

MPS I, MPS1-S, MPS1-HS Trial in Porto Alegre, London, Salford (SIG-005 (hIDUA Producing Spheres))

Withdrawn
  • MPS I
  • +2 more
  • SIG-005 (hIDUA Producing Spheres)
  • Porto Alegre, Brazil
  • +2 more
Dec 16, 2022

Mucopolysaccharidosis I Trial in Wuhan (Laronidase)

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

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

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

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

    Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis VI Trial in Torrance (Adalimumab Injection [Humira],

    Recruiting
    • Mucopolysaccharidosis I
    • +2 more
    • Adalimumab Injection [Humira]
    • Saline Solution for Injection
    • Torrance, California
      The Lundquist Institute at Harbor-UCLA Medical Center
    Aug 9, 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

    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

    MPS IIIA, Sanfilippo Syndrome, Sanfilippo A Trial in Pittsburgh, North Adelaide, Santiago De Compostela (ABO-102)

    Terminated
    • MPS IIIA
    • +3 more
    • Pittsburgh, Pennsylvania
    • +2 more
    Jan 11, 2023

    MPS (RaDiCo Cohort) (RaDiCo-MPS)

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

      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

      MPS IIIA, Sanfilippo Syndrome, Sanfilippo A Trial in Australia, Spain, United States (ABO-102)

      Active, not recruiting
      • MPS IIIA
      • +3 more
      • ABO-102
      • Columbus, Ohio
      • +4 more
      Nov 8, 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

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

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

      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

      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 I, II, and VI Screening inHigh-Risk

      Terminated
      • Mucopolysaccharidosis (MPS)
        • Los Angeles, California
        • +13 more
        Mar 16, 2021

        Mucopolysaccharidosis I, Cognitive Decline Trial in Torrance (Intrathecal recombinant human alpha iduronidase)

        Terminated
        • Mucopolysaccharidosis I
        • Cognitive Decline
        • Intrathecal recombinant human alpha iduronidase
        • Torrance, California
          Los Angeles Biomedical Research Institute at Harbor-UCLA Medical
        Oct 5, 2020

        Mucopolysaccharidosis Type VI Trial in Naples, Ankara (AAV2/8.TBG.hARSB)

        Active, not recruiting
        • Mucopolysaccharidosis Type VI
        • AAV2/8.TBG.hARSB
        • Naples, Italy
        • +1 more
        Nov 22, 2021

        Mucopolysaccharidosis Type IIIA Trial in Manchester (Autologous CD34+ cells transduced with a lentiviral vector containing the

        Active, not recruiting
        • Mucopolysaccharidosis Type IIIA
        • Autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene
        • Manchester, United Kingdom
          Manchester University NHS Foundation Trust
        Jul 2, 2021