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Metachromatic leukodystrophy (MLD)

Showing 1 - 25 of 103
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Lysosomal Storage Diseases, Metachromatic Leukodystrophy Trial in Milan (OTL-200)

Recruiting
  • Lysosomal Storage Diseases
  • Metachromatic Leukodystrophy
  • OTL-200
  • Milan, Italy
    Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR
Nov 16, 2022

Learn About Metachromatic Leukodystrophy (MLD) in Spain

Not yet recruiting
  • Metachromatic Leukodystrophy (MLD)
  • No Intervention
  • Santiago de Compostela, A Coruna, Spain
  • +9 more
Mar 9, 2023

Lysosomal Storage Disease, Metachromatic Leukodystrophy Trial in Milan (OTL-200 Gene Therapy)

Active, not recruiting
  • Lysosomal Storage Disease
  • Metachromatic Leukodystrophy
  • OTL-200 Gene Therapy
  • Milan, Italy
    Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR
Nov 4, 2022

Lysosomal Storage Disease, Metachromatic Leukodystrophy Trial in Milan (OTL-200)

Active, not recruiting
  • Lysosomal Storage Disease
  • Metachromatic Leukodystrophy
  • OTL-200
  • Milan, Italy
    Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR
Sep 29, 2021

Metachromatic Leukodystrophy (MLD) Trial in Hvidovre (rhASA - Dose Level 1, rhASA - Dose Level 2, rhASA - Dose Level 3)

Completed
Has Results
  • Metachromatic Leukodystrophy (MLD)
  • rhASA - Dose Level 1
  • +2 more
  • Hvidovre, Denmark
    Rigshospitalet
Jun 23, 2021

Metachromatic Leukodystrophy (MLD) Trial in Worldwide (SHP611)

Active, not recruiting
  • Metachromatic Leukodystrophy (MLD)
  • Torrance, California
  • +29 more
Dec 13, 2022

Macrophages Activation Pattern in X-linked Adrenoleukodystrophy,

Recruiting
  • Adrenoleukodystrophy
  • +3 more
  • Blood sample collection
  • Le Kremlin-Bicêtre, France
  • +1 more
Oct 10, 2022

Children With Metachromatic Leukodystrophy

Terminated
  • Lipid Metabolism Disorders
  • +13 more
    • Torrance, California
    • +13 more
    Mar 16, 2021

    Metachromatic Leukodystrophy, Adrenoleukodystrophy Trial in Shenzhen (transduced CD34+ hematopoietic stem cell)

    Recruiting
    • Metachromatic Leukodystrophy
    • Adrenoleukodystrophy
    • transduced CD34+ hematopoietic stem cell
    • Shenzhen, Guangdong, China
      Shenzhen Second People's Hospital, The First Affiliated Hospital
    May 25, 2022

    Metachromatic Leukodystrophy Trial in Le Kremlin-Bicêtre (intracerebral administration of AAVrh.10cuARSA)

    Active, not recruiting
    • Metachromatic Leukodystrophy
    • intracerebral administration of AAVrh.10cuARSA
    • Le Kremlin-Bicêtre, France
      Bicêtre Hospital - Paris Sud
    Jan 10, 2022

    Metachromatic Leukodystrophy Study (HOME Study)

    Recruiting
    • Metachromatic Leukodystrophy
      • Danbury, Connecticut
        National Organization for Rare Disorders
      Oct 28, 2021

      Metachromatic Leukodystrophy (MLD) Trial in Shenzhen (Lentivirus-mediated delivery of ARSA to the CNS.)

      Unknown status
      • Metachromatic Leukodystrophy (MLD)
      • Lentivirus-mediated delivery of ARSA to the CNS.
      • Shenzhen, Guangdong, China
        Lung-Ji Chang
      Sep 18, 2019

      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

      Biomarker for Metachromatic Leukodystrophy (BioMeta) Disease

      Active, not recruiting
      • Peripheral Neuropathy
      • Muscle Weakness
        • Cairo, Egypt
        • +4 more
        May 12, 2021

        Metachromatic Leukodystrophy Trial in Durham (Enriched Hematopoetic Stem Cell Infusion)

        No longer available
        • Metachromatic Leukodystrophy
        • Enriched Hematopoetic Stem Cell Infusion
        • Durham, North Carolina
          Duke University
        Jul 15, 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

        Leukodystrophy, White Matter Disease, Leukoencephalopathies Trial in United States

        Recruiting
        • Leukodystrophy
        • +21 more
          • Palo Alto, California
          • +8 more
          Dec 21, 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

          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

          Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

          Recruiting
          • Primary Immunodeficiency (PID)
          • +4 more
          • data collection
          • Pittsburgh, Pennsylvania
            UPMC Children's Hospital of Pittsburgh
          Feb 9, 2022

          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

          Metachromatic Leukodystrophy Trial in Durham (Enriched Hematopoetic Stem Cell Infusion)

          No longer available
          • Metachromatic Leukodystrophy
          • Enriched Hematopoetic Stem Cell Infusion
          • Durham, North Carolina
            Duke University
          Oct 6, 2020

          Inherited Metabolic Disorders Who Underwent Hematopoietic Stem

          Terminated
          • Inherited Metabolic Disorders (IMD)
          • Safety and efficacy assessments
          • Minneapolis, Minnesota
            University of Minnesota
          Mar 10, 2021

          Leukodystrophy: An Observational Study at Sohag University

          Not yet recruiting
          • Children With Leukodystrophy
          • MRI
          • (no location specified)
          Mar 2, 2021

          Hurler Syndrome, Inherited Metabolic Disorder, Lysosomal Storage Disorder Trial in Los Angeles, Palo Alto (rivogenlecleucel,

          No longer available
          • Hurler Syndrome
          • +4 more
          • Los Angeles, California
          • +1 more
          Oct 1, 2020