林业发展局批准Ctexli为首次治疗稀有遗传疾病 -- -- 脑突变性异形病的治疗。 FDA approves Ctexli as the first treatment for the rare genetic disorder, cerebrotendinous xanthomatosis.

林业发展局已核准Ctexli(chenodiol)为首次治疗脑肿瘤Xanthomatis(CTX),这是一种罕见的遗传障碍。 The FDA has approved Ctexli (chenodiol) as the first treatment for cerebrotendinous xanthomatosis (CTX), a rare genetic disorder. 由基因突变引起的CTX导致各种器官中的胆固醇累积。 CTX, caused by a gene mutation, leads to cholesterol buildup in various organs. Ctexli通过替换不足的Bile酸和减少胆固醇沉积来提供帮助。 Ctexli helps by replacing deficient bile acids and reducing cholesterol deposits. 常见的副作用包括腹泻、头痛和腹部疼痛。 Common side effects include diarrhea, headache, and abdominal pain. 批准的依据是试验结果,表明病人情况有显著改善。 This approval is based on trial results showing significant improvement in patients.