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Scn2a

All articles tagged with #scn2a

Low-cost levocarnitine shows autism benefits in gene-defined subset, Yale study finds
health1 day ago

Low-cost levocarnitine shows autism benefits in gene-defined subset, Yale study finds

Yale researchers screened 774 FDA-approved drugs for autism-like traits in genetically modified zebrafish, narrowing to 520 non-toxic candidates; levocarnitine (Carnitor), a cheap prescription drug, stood out by reversing disruptive behaviors and normalizing brain activity in zebrafish with SCN2A and DYRK1A mutations, genes linked to autism. Levocarnitine costs about 44 cents per pill and is approved for carnitine deficiency, but these two mutations are rare in the broader autism population, so benefits would likely apply to a small subset. The work is preclinical, and researchers caution against using levocarnitine to treat autism until human trials are conducted; they also created an open-source drug database to guide future research.