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FDA Approves First Treatment for Cerebrotendinous Xanthomatosis, a Rare Lipid Storage Disease
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U.S. Food and Drug Administration, Mirum Pharmaceuticals Inc.  
February 21, 2025

FDA Approves First Treatment for Cerebrotendinous Xanthomatosis, a Rare Lipid Storage Disease

SILVER SPRING, Md., Feb. 21, 2025 /PRNewswire/ -- Today, the U.S. Food and Drug Administration approved Ctexli (chenodiol) for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Ctexli is the

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