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Niemann-pick type C,

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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

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

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

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

    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

    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

    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

      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

      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

        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

        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 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

          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

          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 C Trial (VTS-270)

          Withdrawn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

          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

          Niemann-Pick Disease, Type C Trial in Worldwide (arimoclomol, Placebo)

          Active, not recruiting
          • Niemann-Pick Disease, Type C
          • Oakland, California
          • +13 more
          Apr 14, 2021

          Pediatric Patients With Unexplained Enlarged Spleen

          Recruiting
          • Gaucher Disease, Splenomegaly
          • Acid SphingoMyelinase Deficiency
            • Amiens, France
            • +39 more
            Aug 19, 2022