FDA ap­proves first drug to treat ul­tra-rare lipid stor­age dis­ease

The FDA on Fri­day ap­proved Mirum Phar­ma­ceu­ti­cals’ Ctexli to treat adults with cere­bro­tendi­nous xan­thomato­sis (CTX), a rare, pro­gres­sive and ge­net­ic dis­ease.

The ap­proval is based …

Sign up to read this article for free.

Get free access to a limited number of articles, plus choose newsletters to get straight to your inbox.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER