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The Myelin Disorders Biorepository Project

Join our Myelin Disorders Biorepository Project to help advance research!

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The Myelin Disorders Biorepository Project (MDBP) collects and studies samples from people with leukodystrophy, a group of rare brain disorders. These disorders affect the white matter in the brain, which is like insulation for brain cells. The project aims to help researchers find new causes for these disorders, create tools to help diagnose them, and learn more about how they develop over time.

In the past, diagnosing these disorders was difficult and expensive. New methods like whole exome sequencing, which looks at a person's genes, are improving diagnosis rates. However, about one-third of patients still don't receive a clear diagnosis. This research hopes to change that by finding new information and improving care and treatment options for people with leukodystrophies.

  • The study is open to all ages with suspected or confirmed leukodystrophy, or healthy individuals without these disorders.
  • Participants must give consent and be willing to share medical data and samples.
  • The project is ongoing, with no specific end date mentioned.
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

13 December 2024

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