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Mucopolysaccharidosis I

Showing 1 - 25 of >10,000
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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 I Trial in Wuhan (Laronidase)

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

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

Completed
  • Mucopolysaccharidosis I
  • Oakland, California
  • +5 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

Long Term Follow-Up for RGX-111

Enrolling by invitation
  • Mucopolysaccharidosis I
  • No Intervention
  • Orange, California
  • +2 more
Oct 25, 2023

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

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

MPS (RaDiCo Cohort) (RaDiCo-MPS)

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

    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 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 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 III-A Trial in Hamburg (JR-441)

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

    (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX

    Active, not recruiting
    • Hemophilia B
    • +2 more
    • SB-318
    • +2 more
    • Oakland, California
    • +4 more
    Jan 30, 2023

    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

    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, 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

      Examinations for Asymptomatic MPS I Infants in Taiwan

      Recruiting
      • MPS I
      • MPS I
      • Taipei, Taiwan
        Mackay Memorial Hospital
      Mar 2, 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 II Trial in Japan (JR-141)

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

      Patients Who Have Received Gene Therapy Or Gene Marked Products

      Recruiting
      • Severe Combined Immunodeficiency
      • +4 more
      • Venipuncture
      • Memphis, Tennessee
        St. Jude Children's Research Hospital
      Dec 10, 2021

      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 IH Trial in Milano (Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified

      Active, not recruiting
      • Mucopolysaccharidosis IH
      • Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LV, encoding for the α-L-iduronidase cDNA, in their final formulation medium.
      • Milano, Italy
        Ospedale San Raffaele
      Aug 6, 2021

      Baby Detect : Genomic Newborn Screening

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

        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