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Mirum’s CTEXLI™ (chenodiol) Tablets Receives FDA Approval for Treatment of Cerebrotendinous Xanthomatosis (CTX)
CTEXLI is the first and only medication approved for the treatment of CTX in adults Approval based on Phase 3 RESTORE study results CTEXLI granted U.S. FDA Orphan Drug exclusivity for the ...
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FDA Breakthrough: First-Ever Treatment Approved for Rare and Devastating CTX Disorder!
CTX stems from a genetic mutation in the CYP27A1 gene, resulting in a deficiency of an enzyme essential for lipid breakdown. This deficiency disrupts bile acid production in the liver, preventing ...
Business Wire26d
Mirum’s CTEXLI™ (chenodiol) Tablets Receives FDA Approval for Treatment of Cerebrotendinous Xanthomatosis (CTX)
The approval is based on data from the Phase 3 RESTORE study evaluating the safety and efficacy of CTEXLI in adult patients with CTX by measurement of urine bile alcohols and other secondary measures.