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  2. Canavan disease - Wikipedia

    en.wikipedia.org/wiki/Canavan_disease

    Symptoms of the most common (and most serious) form of Canavan disease typically appear in early infancy usually between the first three to six months of age. [4] Canavan disease then progresses rapidly from that stage, with typical cases involving intellectual disability, loss of previously acquired motor skills, feeding difficulties, abnormal muscle tone (i.e., initial floppiness ...

  3. Spongy degeneration of the central nervous system - Wikipedia

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

    The first case of spongy degeneration of the CNS was reported in 1928 by Globus and Strauss, [42] who designated the case as Schilder's disease, a term for diffuse myelinoclastic sclerosis. [43] [44] [45] In 1931, Canavan reported a case where the megalencephaly of brain degeneration is different from that caused by a tumour. [46]

  4. 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).

  5. 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.

  6. Aspartoacylase - Wikipedia

    en.wikipedia.org/wiki/Aspartoacylase

    Canavan disease is a rare autosomal recessive disorder that causes spongy degeneration of the white matter in the brain and severe psychomotor retardation, usually leading to death at a young age. [ 12 ] [ 20 ] The loss of aspartoacylase activity leads to the buildup of N-acetyl-L-aspartate in the brain and an increase in urine concentration by ...

  7. Dentate nucleus - Wikipedia

    en.wikipedia.org/wiki/Dentate_nucleus

    Glutaric aciduria type 1 (GA1): An autosomal recessive disease, GA1 is due to glutaryl-coenzyme A dehydrogenase deficiency. Abnormalities are seen in the basal ganglia and dentate nucleus. [12] Canavan's disease: Canavan's disease is a white matter disease due to aspartoacylase deficiency. The dentate nucleus is not affected until late in ...

  8. Maps show Helene's path as it batters Florida and Southeast - AOL

    www.aol.com/maps-show-track-potential-hurricane...

    Maps show the areas impacted by storm surge, rainfall levels and more as Helene, once a major hurricane and now a tropical storm, moves inland from Florida's Gulf Coast over Georgia.

  9. Myrtelle Canavan - Wikipedia

    en.wikipedia.org/wiki/Myrtelle_Canavan

    Myrtelle May Moore Canavan [1] (June 24, 1879 – August 4, 1953) was an American physician and medical researcher. She was one of the first female pathologists and is best known for publishing a description of Canavan disease in 1931.