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Hereditary Leukodystrophy with Spheroids (CSF1R-related HLDS),

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

ALSP Trial in Englewood, Jacksonville (VGL101)

Recruiting
  • ALSP
  • Englewood, Colorado
  • +1 more
Dec 22, 2022

Macrophages Activation Pattern in X-linked Adrenoleukodystrophy,

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

Ultra-rare Inherited Metabolic and Degenerative Neurological

Recruiting
  • Inherited Disease
  • +5 more
  • collection of data
  • Milano, Italy
    Fondazione IRCCS Istituto Neurologico Carlo Besta
May 6, 2021

Adult-Onset Leukoencephalopathy With Axonal Spheroids and

Recruiting
  • ALSP
  • No intervention
  • San Francisco, California
  • +7 more
May 13, 2022

Leukodystrophy, White Matter Disease, Leukoencephalopathies Trial in United States

Recruiting
  • Leukodystrophy
  • +21 more
    • Palo Alto, California
    • +8 more
    Dec 21, 2021

    Metachromatic Leukodystrophy (MLD) Trial in Worldwide (SHP611)

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

    Phenotype, Genotype and Biomarkers 2

    Recruiting
    • Amyotrophic Lateral Sclerosis
    • +4 more
      • Miami, Florida
      • +3 more
      Dec 19, 2022

      Positron Emission Tomography, Alzheimer, Neurodegenerative Diseases Trial in Shanghai ([18F]CSF-23)

      Recruiting
      • Positron Emission Tomography
      • +2 more
      • [18F]CSF-23
      • Shanghai, China
        Huashan Hospital
      Nov 19, 2023

      Retinitis Pigmentosa, Stem Cell Transplant Trial in Istanbul (Stem cell implantation)

      Completed
      • Retinitis Pigmentosa
      • Stem Cell Transplant
      • Stem cell implantation
      • Istanbul, Turkey
        Acıbadem University
      Jan 25, 2023

      Assess CSF1R-related Leukoencephalopathy After Stem Cell

      Enrolling by invitation
      • CSF1R-related Leukoencephalopathy
      • +3 more
        • Jacksonville, Florida
          Mayo Clinic in Florida
        Dec 7, 2021

        Effort Test asTherapeutic Monitoring Tool in Acute

        Completed
        • Rhabdomyolysis Linked to a Hereditary Disease of Metabolism
        • Effort test
        • Functional tests on fibroblasts
        • Paris, France
          Hôpital Necker-Enfants Malades
        Oct 10, 2022

        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

        Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy,

        Recruiting
        • Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
        • +2 more
        • NTLA-2001
        • Paris, France
        • +3 more
        Nov 29, 2022

        Head and Neck Squamous Cell Carcinoma Trial in Baltimore (Nivolumab, HuMax-IL8, Cabiralizumab)

        Recruiting
        • Head and Neck Squamous Cell Carcinoma
        • Baltimore, Maryland
          Johns Hopkins University
        Jul 28, 2022

        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

        Hereditary Hemochromatosis, Hyperferritinemia Trial in Basel, Berne (Venipuncture)

        Enrolling by invitation
        • Hereditary Hemochromatosis
        • Hyperferritinemia
        • Venipuncture
        • Basel, Switzerland
        • +1 more
        Feb 14, 2023

        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

        Subfertility Trial in Hong Kong (endometrial biopsy)

        Completed
        • Subfertility
        • endometrial biopsy
        • Hong Kong, Hong Kong, China
          Department of Obstetrics and Gynaecology
        Mar 2, 2022

        Hereditary Hemorrhagic Telangiectasia, HHT, Rendu Osler Weber Disease Trial in Madrid (Registry)

        Recruiting
        • Hereditary Hemorrhagic Telangiectasia
        • +2 more
        • Registry
        • Madrid, Spain
          Hospital Universitario Ramón y Cajal
        Sep 30, 2022

        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

        Quality Trial in Palo Alto (Educational video and pamphlet, Traditional genetic counseling)

        Recruiting
        • Quality
        • Educational video and pamphlet
        • Traditional genetic counseling
        • Palo Alto, California
          Stanford University
        Jan 25, 2023

        Leukodystrophy, Globoid Cell Trial in Worldwide (PBKR03)

        Recruiting
        • Leukodystrophy, Globoid Cell
        • PBKR03
        • Chicago, Illinois
        • +8 more
        Aug 17, 2022

        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

        Hereditary Spastic Paraplegia, Neurodegenerative Diseases, Pediatric Disorder Trial in Boston

        Recruiting
        • Hereditary Spastic Paraplegia
        • +5 more
          • Boston, Massachusetts
            Boston Children's Hospital
          Aug 5, 2022