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The Myelin Disorders Biorepository Project

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The Myelin Disorders Biorepository Project is a research study that collects clinical data and biological samples from people with leukodystrophy, a rare genetic disorder affecting the brain's white matter. The study aims to discover new causes of these disorders, find better diagnostic tools, and improve future treatments. Leukodystrophies are tricky to diagnose, often taking years and lots of testing, costing patients a lot of money. Researchers use advanced genetic testing to improve diagnosis rates and understand these disorders better.

Eligibility: People of any age with or without leukodystrophy can participate. Those with leukodystrophy should have a suspected or confirmed diagnosis, while healthy controls should not have any diagnosis. All participants need to provide informed consent.

  • Participation involves sharing clinical data and biological samples.
  • Individuals will be contacted for future research opportunities.
  • No specific treatment is offered; the study is for research purposes only.

Participation supports important research that may lead to new discoveries and better care for those with leukodystrophy. 🌟

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Study details
    Leukodystrophy
    White Matter Disease
    Leukoencephalopathies
    4H Syndrome
    Adrenoleukodystrophy
    AMN
    ALD
    ALD Gene Mutation
    ALD (Adrenoleukodystrophy)
    X-linked Adrenoleukodystrophy
    X-ALD
    Adrenomyeloneuropathy
    Aicardi Goutieres Syndrome
    AGS
    Alexander Disease
    Alexanders Leukodystrophy
    AxD
    ADLD
    Canavan Disease
    CTX
    Cerebrotendinous Xanthomatoses
    Krabbe Disease
    GALC Deficiency
    Globoid Leukodystrophy
    TUBB4A-Related Leukodystrophy
    H-ABC - Hypomyelination
    Atrophy of Basal Ganglia and Cerebellum
    HBSL
    HBSL - Hypomyelination
    Brain Stem
    Spinal Cord
    Leg Spasticity
    LBSL
    Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder)
    Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation
    ALSP
    CSF1R Gene Mutation
    HCC - Hypomyelination and Congenital Cataract
    MLC1
    Megalencephalic Leukoencephalopathy With Subcortical Cysts
    MLD
    Metachromatic Leukodystrophy
    PMD
    Pelizaeus-Merzbacher Disease
    PLP1 Null Syndrome
    PLP1 Gene Duplication | Blood or Tissue | Mutations
    Pelizaeus Merzbacher Like Disease
    Peroxisomal Biogenesis Disorder
    Zellweger Syndrome
    Refsum Disease
    Salla Disease
    Sialic Storage Disease
    Sjögren
    Sjogren-Larsson Syndrome
    Van Der Knapp Disease
    Vanishing White Matter Disease
    Charcot-Marie-Tooth
    CMT
    Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency
    Allan-Herndon-Dudley Syndrome
    Cadasil
    Cockayne Syndrome
    Multiple Sulfatase Deficiency
    Gangliosidoses
    GM2 Gangliosidosis
    BPAN
    Labrune Syndrome
    LCC
    Mucopolysaccharidoses
    TBCK-Related Intellectual Disability Syndrome

NCT03047369

Children's Hospital of Philadelphia

20 June 2024

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