FDA Approves First Drug for Rare Lipid Storage Disorder

Watchdoq February 21, 2025
(MedPage Today) -- The FDA on Friday approved chenodiol (Ctexli) for the treatment of cerebrotendinous xanthomatosis (CTX) in adults.
Chenodiol -- a naturally occurring human bile acid also known as chenodeoxycholic acid -- is the first FDA...

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