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mucopolysaccharidosis

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Mucopolysaccharidosis II Trial in Japan (JR-141)

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

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

Hunter Syndrome Trial in Lexington

Recruiting
  • Hunter Syndrome
    • Lexington, Massachusetts
      Shire
    Nov 7, 2022

    Mucopolysaccharidosis II Trial (Autologous CD34+ HSCs transduced ex vivo with CD11B LV encoding human IDS tagged with ApoEII)

    Not yet recruiting
    • Mucopolysaccharidosis II
    • Autologous CD34+ HSCs transduced ex vivo with CD11B LV encoding human IDS tagged with ApoEII
    • (no location specified)
    Dec 16, 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, 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 Trial in Netherlands, United States (DNL310)

    Recruiting
    • Mucopolysaccharidosis II
    • Oakland, California
    • +4 more
    Nov 14, 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

    MPS I Trial in Oakland (SB-318)

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

    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 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

    Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy Trial in Denver, Valhalla, Salt Lake City (Human

    Completed
    • Mucopolysaccharidosis I
    • +15 more
    • Human Placental Derived Stem Cell
    • Denver, Colorado
    • +2 more
    Oct 24, 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

      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

      MPS II Immunophenotyping

      Recruiting
      • MPS II
      • +2 more
        • Durham, North Carolina
          Duke University
        Jun 3, 2022

        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

        Hunter Syndrome Trial in India (Elaprase)

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

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

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

        Study of Patients With MPS IIIA Treated With ABO-102

        Enrolling by invitation
        • Mucopolysaccharidosis III-A
        • ABO-102
        • Columbus, Ohio
        • +2 more
        Nov 8, 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

        Baby Detect : Genomic Newborn Screening

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

          Patients With Sanfilippo Disease(s) (MPS3)

          Not yet recruiting
          • Sanfilippo Syndrome
          • MPS3
            • Fairfax, Virginia
              LDRTC
            Jan 20, 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