FDA drug alerts in endocrinology, including drug approvals, drug warnings, FDA medical device recalls, and FDA drug label ...

2025 年 2 月 21 日，美国食品药品监督管理局（FDA）批准Mirum Pharmaceuticals旗下Ctexli用于治疗成人脑腱黄瘤病（CTX）。Ctexli是FDA批准的首个用于治疗CTX的药物。FDA罕见病、儿科、泌尿和生殖医学办公室主任Janet ...

Several rare disease patient populations received their first-ever FDA-approved drug since Rare Disease Day last year, ...

ITF, IntraBio and Orchard are among the companies that have won FDA nods in the past year for Duchenne muscular dystrophy, ...

Around the world, events are being held to mark Rare Disease Day (28 February 2025), with the aim of bringing attention to ...

EST Mirum Pharmaceuticals (MIRM) sees FY25 product sales of $420M-$435M vs. $336.4M in FY24Discover the Best Stocks and Maximize Your ...

US Food and Drug Administration (FDA) has approved Ctexli (chenodiol) tablets, a bile acid, for the treatment of adults with cerebrotendinous xanthomatosis (CTX). Ctexli is the first and only ...

The Food and Drug Administration has approved Ctexli (chenodiol) for the treatment of cerebrotendinous xanthomatosis in adults.

In honor of Rare Disease Day, here are 10 new treatments approved by the FDA to treat rare diseases over the past year.

The US Food and Drug Administration (FDA) has approved Mirum Pharmaceuticals’ Ctexli (chenodiol) to treat adults with ...

A young Peninsula drugmaker won regulatory approval Friday for a rare-disease drug picked up in a deal two years ago.

Ctexli’s approval further entrenches Mirum as a leader in rare liver diseases, alongside its cornerstone product Livmarli and upcoming drug volixibat.