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  2. Leukodystrophy - Wikipedia

    en.wikipedia.org/wiki/Leukodystrophy

    The European Leukodystrophy Association also supports research into leukodystrophy. As of 2020, more than 387 research projects have been funded. Each year, ELA invites the international scientific community to submit research projects in the field of genetic leukodystrophies, the cerebral white matter in premature infants, and of myelin repair.

  3. Leukoencephalopathy with vanishing white matter - Wikipedia

    en.wikipedia.org/wiki/Leukoencephalopathy_with...

    T2-weighted images also displaying cerebrospinal fluid and rarefied/cystic white matter. To view the remaining tissue, and get perspective on the damage done (also helpful in determining the rate of deterioration) (T1-weighted), proton density, and FLAIR images are ideal as they show radiating stripe patterns in the degenerating white matter.

  4. Krabbe disease - Wikipedia

    en.wikipedia.org/wiki/Krabbe_disease

    Krabbe disease (KD) (also known as globoid cell leukodystrophy [3] or galactosylceramide lipidosis) is a rare and often fatal lysosomal storage disease that results in progressive damage to the nervous system. KD involves dysfunctional metabolism of sphingolipids and is inherited in an autosomal recessive pattern.

  5. Metachromatic leukodystrophy - Wikipedia

    en.wikipedia.org/wiki/Metachromatic_leukodystrophy

    The incidence of metachromatic leukodystrophy is estimated to occur in 1 in 40,000 to 1 in 160,000 individuals worldwide. [13] There is a much higher incidence in certain genetically isolated populations, such as 1 in 75 in Habbanites (a small group of Jews who immigrated to Israel from southern Arabia), 1 in 2,500 in the western portion of the ...

  6. Retinal vasculopathy with cerebral leukoencephalopathy and ...

    en.wikipedia.org/wiki/Retinal_vasculopathy_with...

    Retinal vasculopathy with cerebral leukocencephalopathy and systemic manifestations (RVCL or RVCL-S, also previously known as retinal vasculopathy with cerebral leukodystrophy, RVCL; or cerebroretinal vasculopathy, CRV; or hereditary vascular retinopathy, HVR; or hereditary endotheliopathy, retinopathy, nephropathy, and stroke, HERNS) is an inherited condition resulting from a frameshift ...

  7. US approves first gene therapy for children with rare genetic ...

    www.aol.com/news/us-approves-first-gene-therapy...

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  8. Cockayne syndrome - Wikipedia

    en.wikipedia.org/wiki/Cockayne_syndrome

    Cockayne syndrome (CS), also called Neill-Dingwall syndrome, is a rare and fatal autosomal recessive neurodegenerative disorder characterized by growth failure, impaired development of the nervous system, abnormal sensitivity to sunlight (photosensitivity), eye disorders and premature aging.

  9. Medical genetics of Jews - Wikipedia

    en.wikipedia.org/wiki/Medical_genetics_of_Jews

    Bloom syndrome: Medical genetics Autosomal recessive BLM: 1/100 Breast cancer and ovarian cancer: Oncology Autosomal dominant BRCA1 or BRCA2: 1/100 and 1/75, respectively Canavan disease: Endocrinology, neurology Autosomal recessive ASPA: 1/60 Congenital deafness: Neurology, otorhinolaryngology, audiology Autosomal recessive GJB2 or GJB6: 1/25