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Niemann-Pick C subtype 2

Showing 1 - 25 of >10,000
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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

GM2 Gangliosidosis, Niemann-Pick Disease, Type C Trial in Curitiba, Porto Alegre, Rio De Janeiro (AZ-3102 (Dose 1), Placebo,

Not yet recruiting
  • GM2 Gangliosidosis
  • Niemann-Pick Disease, Type C
  • AZ-3102 (Dose 1)
  • +2 more
  • Curitiba, Brazil
  • +2 more
Feb 24, 2023

Asmd, Visceral Type Trial in Amsterdam (MR Spectroscopy (MRS), MR Elastography (MRE))

Recruiting
  • Asmd, Visceral Type
  • MR Spectroscopy (MRS)
  • MR Elastography (MRE)
  • Amsterdam, Noord-Holland, Netherlands
    Amsterdam UMC
Jun 6, 2023

Genomic and Phenotypic Database for Niemann-Pick Disease, Type C

Recruiting
  • Niemann-Pick Disease, Type C
    • Bethesda, Maryland
      National Institutes of Health Clinical Center
    Feb 2, 2023

    Niemann-Pick Disease, Type C1 Trial in Worldwide (Hydroxypropyl-beta-cyclodextrin, Placebo)

    Recruiting
    • Niemann-Pick Disease, Type C1
    • Oakland, California
    • +13 more
    Jan 9, 2023

    Niemann-Pick Type C Disease Trial in United States (Adrabetadex)

    Completed
    • Niemann-Pick Type C Disease
    • Little Rock, Arkansas
    • +16 more
    Oct 7, 2022

    Functional Near-Infrared Spectroscopy Combined With Diffuse

    Recruiting
    • Juvenile Neuronal Ceroid Lipofuscinosis
    • +3 more
      • Bethesda, Maryland
        National Institutes of Health Clinical Center
      Jan 30, 2023

      PREDIGA 2: Spanish Acronym of "Educational and Diagnostic

      Not yet recruiting
      • Gaucher Disease
      • +4 more
      • Patient with splenomegaly or splenectomy
      • Barcelona, Spain
        Hospital Vall d'Hebron
      Dec 13, 2022

      Niemann-Pick Disease, Type C Trial in United States (Arimoclomol)

      Available
      • Niemann-Pick Disease, Type C
      • Birmingham, Alabama
      • +14 more
      Aug 18, 2022

      Sphingomyelin Lipidosis Trial (olipudase alfa (GZ402665))

      Approved for marketing
      • Sphingomyelin Lipidosis
      • olipudase alfa (GZ402665)
      • (no location specified)
      Sep 15, 2022

      Niemann-Pick Disease, Type C1 Trial run by the (VTS-270)

      Completed
      • Niemann-Pick Disease, Type C1
      • Bethesda, Maryland
        National Institutes of Health Clinical Center, 9000 Rockville Pi
      Jul 19, 2021

      Biomarker for Niemann Pick Type C Disease (BioNPC)

      Active, not recruiting
      • Niemann-Pick Disease
      • Niemann-Pick Disease, Type C
        • Rostock, Germany
        • +3 more
        May 12, 2021

        Acid Sphingomyelinase Deficiency Disease and Gaucher Disease in

        Recruiting
        • Gaucher Disease
        • Acid SphingoMyelinase Deficiency
          • Ponferrada, León, Spain
          • +11 more
          Aug 8, 2023

          Adult and Pediatric Acid Sphingomyelinase Deficiency on Early

          Recruiting
          • Acid Sphingomyelinase Deficiency (ASMD)
          • Olipudase alfa
          • France, France
            Investigational site number France
          Jun 13, 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

          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

          Niemann-Pick Disease, Type C1 Trial in Oakland (Hydroxypropyl-ß-cyclodextrin)

          Active, not recruiting
          • Niemann-Pick Disease, Type C1
          • Oakland, California
            UCSF Benioff Children's Hospital Oakland
          Jun 16, 2021

          Complement Activation in the Lysosomal Storage Disorders

          Withdrawn
          • Fabry Disease
          • +3 more
          • Complement measurements
          • Melbourne, Victoria, Australia
            The Royal Melbourne Hospital
          Jun 2, 2021

          Niemann-Pick Disease, Type C Trial in Worldwide (Parts A/B: Adrabetadex, Parts A/B: Sham Control)

          Active, not recruiting
          • Niemann-Pick Disease, Type C
          • Parts A/B: Adrabetadex
          • Parts A/B: Sham Control
          • Birmingham, Alabama
          • +28 more
          Jul 23, 2021

          Sphingomyelin Lipidosis Trial in Worldwide (Olipudase alfa)

          Completed
          Has Results
          • Sphingomyelin Lipidosis
          • Olipudase alfa
          • New York, New York
          • +5 more
          Mar 15, 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

          Niemann-Pick Disease, Type C1 Trial in Oakland, Morristown (Hydroxypropyl-beta-cyclodextrin)

          Completed
          • Niemann-Pick Disease, Type C1
          • Oakland, California
          • +1 more
          Feb 17, 2021

          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

          Health Insurance Literacy and Challenges in Accessing Health

          Completed
          • Niemann-Pick Diseases
            • New York, New York
              Icahn School of Medicine at Mount Sinai
            Oct 5, 2020

            Niemann-Pick Disease, Type C Trial (VTS-270)

            Withdrawn
            • Niemann-Pick Disease, Type C
            • (no location specified)
            Jul 20, 2020