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  2. Canavan disease - Wikipedia

    en.wikipedia.org/wiki/Canavan_disease

    Canavan disease, or Canavan–Van Bogaert–Bertrand disease, is a rare and fatal autosomal recessive [1] degenerative disease that causes progressive damage to nerve cells and loss of white matter in the brain. [2] It is one of the most common degenerative cerebral diseases of infancy. [3]

  3. Spongy degeneration of the central nervous system - Wikipedia

    en.wikipedia.org/wiki/Spongy_degeneration_of_the...

    Canavan's disease is initially recognized by the appearance of symptoms, yet further examinations are needed for definitive diagnosis. Neuroimaging techniques such as Computed Tomography (CT) scan or Magnetic Resonance imaging (MRI) are typically used to detect the presence of degenerative subcortical white matter . [ 26 ]

  4. BridgeBio Shares Positive Data from High Dose Cohort of Phase ...

    lite.aol.com/tech/story/0022/20241024/9261193.htm

    About Canavan Disease Affecting approximately 1,000 children in the U.S. and European Union, Canavan disease is an ultra-rare, disabling and fatal disease with no approved therapy. Most children are not able to meet developmental milestones, are unable to crawl, walk, sit or talk, and die at a young age.

  5. Leukodystrophy - Wikipedia

    en.wikipedia.org/wiki/Leukodystrophy

    Canavan disease is a less-studied type of leukodystrophy that, like MLD and Krabbe disease, is also inherited in an autosomal recessive pattern. It is due to a mutation in the ASPA gene that encodes aspartoacylase , an enzyme needed to metabolize N-acetyl-L-aspartate (NAA).

  6. 'Before and After Zachariah': What the story means 40 ... - AOL

    www.aol.com/zachariah-story-means-40-years...

    Zachariah, born in 1976, had Canavan disease, a rare leukodystrophy syndrome that causes brain deterioration. There were years going to doctors and therapists, searching for answers.

  7. BridgeBio Receives FDA’s Regenerative Medicine Advanced ...

    lite.aol.com/tech/story/0022/20240910/9228699.htm

    About Canavan Disease Affecting approximately 1,000 children in the U.S. and European Union, Canavan disease is an ultra-rare, disabling and fatal disease with no approved therapy. Most children are not able to meet developmental milestones, are unable to crawl, walk, sit or talk, and die at a young age.

  8. Medical genetics of Jews - Wikipedia

    en.wikipedia.org/wiki/Medical_genetics_of_Jews

    The CIGNA panel is available for testing for parental/preconception screening or following chorionic villus sampling or amniocentesis and tests for Bloom syndrome, Canavan disease, cystic fibrosis, familial dysautonomia, Fanconi anemia, Gaucher disease, mucolipidosis IV, Neimann-Pick disease type A, Tay-Sachs disease, and torsion dystonia.

  9. 111 Of The Rarest Genetic Mutations Ever That People ... - AOL

    www.aol.com/111-rarest-genetic-mutations-ever...

    “Mr. Schubarth's actions not only violated multiple laws designed to protect wildlife, but also risked introducing diseases and compromising the genetic integrity of our wild sheep populations ...