Phase II Study of Cholesterol- and Cholestanol-Free Diet, Lovastatin, and Chenodeoxycholic Acid for Cerebrotendinous Xanthomatosis

计划入组 5 人 · 首次发布于 1999 年 10 月 19 日

Cholestanol in Humans

首次发布于 2001 年 7 月 5 日

The Prevalence of CTX Disorder in Juvenile Cataract Cases in Turkey

计划入组 1,000 人 · 首次发布于 2018 年 7 月 12 日

FDA Approves First Treatment for Cerebrotendinous Xanthomatosis: Mirum's Chenodiol Tablets

FDA Approves First Treatment for Cerebrotendinous Xanthomatosis (CTX), Offering Hope to Patients with Rare Genetic Disorder

FDA Approves Five Groundbreaking Treatments for Ultra-Rare Diseases in Recent Months