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  2. Leukodystrophy - Wikipedia

    en.wikipedia.org/wiki/Leukodystrophy

    Leukodystrophy is characterized by specific symptoms, including decreased motor function, muscle rigidity, and eventual degeneration of sight and hearing. While the disease is fatal, the age of onset is a key factor, as infants have a typical life expectancy of 2–8 years, while adults typically live more than a decade after onset.

  3. Leukoencephalopathy with vanishing white matter - Wikipedia

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

    Childhood ataxia with central nervous system hypomyelinization, Vanishing white matter leukodystrophy, Cree leukoencephalopathy, Vanishing white matter leukodystrophy with ovarian failure, included, Myelinopathia centralis diffusa: This condition is inherited in an autosomal recessive manner

  4. Autosomal dominant leukodystrophy with autonomic disease

    en.wikipedia.org/wiki/Autosomal_dominant...

    Autosomal dominant leukodystrophy with autonomic disease is a rare neurological condition of genetic origin which is ... the life expectancy can't be improved easily ...

  5. Metachromatic leukodystrophy - Wikipedia

    en.wikipedia.org/wiki/Metachromatic_leukodystrophy

    The drug became a source of controversy when a family attempted to purchase the drug for their child before it was approved. [34] Jonckheere, Kingma, Eyskens, Bordon, & Jansen (2023) highlight the shift towards the need for newborn screening for metachromatic leukodystrophy as it allows for improved early detection and timely treatment as well ...

  6. Krabbe disease - Wikipedia

    en.wikipedia.org/wiki/Krabbe_disease

    The mortality rate of early infantile Krabbe disease is 90% before age two. Later onset of symptoms is associated with longer life expectancy, with older children generally surviving two to seven years after the initial diagnosis. [22] Krabbe disease occurs in about one in 100,000 births. [23]

  7. $4.25 million gene therapy for kids becomes world's priciest drug

    www.aol.com/4-25-million-gene-therapy-025114680.html

    A lifesaving gene therapy for children born with a rare and debilitating disease has just been approved by the U.S. Food and Drug Administration.The catch? Its wholesale cost has been set at $4.25 ...

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

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

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  9. Alexander disease - Wikipedia

    en.wikipedia.org/wiki/Alexander_disease

    Alexander disease is a very rare autosomal dominant leukodystrophy, which are neurological conditions caused by anomalies in the myelin which protects nerve fibers in the brain. The most common type is the infantile form that usually begins during the first two years of life.

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