MedPage Today11h
FDA Approves First Drug for Rare Lipid Storage Disorder
Chenodiol -- a naturally occurring human bile acid also known as chenodeoxycholic acid -- is the first FDA-approved drug ...
Medindia4h
FDA Approves First Treatment for Cerebrotendinous Xanthomatosis, a Rare Lipid Storage Disease
"The FDA is dedicated to supporting new drug development for rare diseases including very rare metabolic diseases like cerebrotendinous xanthomatosis," said Janet Maynard, M.D., M ...