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Mucopolysaccharidosis II

Showing 26 - 50 of >10,000
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Hunter Syndrome Trial (Idursulfase-IT)

Available
  • Hunter Syndrome
  • (no location specified)
Apr 6, 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, Hunter Syndrome Trial in Pusan, Seoul (GC1123)

Not yet recruiting
  • Mucopolysaccharidosis II
  • Hunter Syndrome
  • GC1123
  • Pusan, Korea, Republic of
  • +2 more
Jun 16, 2022

Adrenoleukodystrophy, Batten Disease, Mucopolysaccharidosis II Trial in Durham (DUOC-01)

Recruiting
  • Adrenoleukodystrophy
  • +11 more
  • DUOC-01
  • Durham, North Carolina
    Duke University Medical Center
Oct 24, 2022

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

Completed
  • Mucopolysaccharidosis I
  • Oakland, California
  • +5 more
Aug 16, 2022

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

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

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

Hunter Syndrome Trial in Canada, France (Idursulfase-IT, Elaprase)

Not yet recruiting
  • Hunter Syndrome
  • Calgary, Alberta, Canada
  • +3 more
Sep 4, 2023

Baby Detect : Genomic Newborn Screening

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

    Long-term Follow-Up for RGX-121

    Enrolling by invitation
    • Mucopolysaccharidosis II
    • Long-term Follow-Up
    • Pittsburgh, Pennsylvania
      Children's Hospital of Pittsburgh - UPMC: Program for Neurodevel
    Aug 9, 2021

    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 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

    Mucopolysaccharidosis II Trial in Worldwide (Iduronate-2-sulfatase enzyme replacement therapy, iduronate-2-sulfatase enzyme

    Completed
    Has Results
    • Mucopolysaccharidosis II
    • Iduronate-2-sulfatase enzyme replacement therapy
    • +2 more
    • Oakland, California
    • +8 more
    May 30, 2021

    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

    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

      Biomarkers for Hunter Syndrome

      Active, not recruiting
      • Hunter Syndrome
      • +3 more
        • Cancun, Quintana Roo, Mexico
        • +3 more
        May 12, 2021

        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

        Hunter Syndrome, Mucopolysaccharidosis II, MPS II Trial in Porto Alegre, Warsaw, Taipei (Idursulfase)

        Completed
        Has Results
        • Hunter Syndrome
        • +2 more
        • Idursulfase
        • Porto Alegre, RS, Brazil
        • +2 more
        May 14, 2021

        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

        Hunter Syndrome Trial in Worldwide (Idursulfase-IT, Elaprase)

        Active, not recruiting
        • Hunter Syndrome
        • Oakland, California
        • +8 more
        Aug 31, 2022

        Neurodevelopmental Status in Pediatric Hunter Syndrome (MPS II)

        Completed
        • Mucopolysaccharidosis (MPS)
        • Hunter Syndrome
          • Oakland, California
          • +6 more
          Mar 15, 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

          Identify Pediatric Hunter Syndrome Who Demonstrate Evidence of

          Completed
          Has Results
          • Hunter Syndrome
          • Neurobehavioral testing
          • Visual and auditory assessments
          • Chapel Hill, North Carolina
          • +1 more
          May 18, 2021