Chenodiol -- a naturally occurring human bile acid also known as chenodeoxycholic acid -- is the first FDA-approved drug indicated for the rare lipid storage disease and autosomal recessive genetic ...
"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 ...
The U.S. Food and Drug Administration approved Mirum Pharmaceuticals' genetic disorder drug on Friday, making it the first ...
On Monday, the Scottsboro City Council held a work session. There were seven items on the work session agenda, with five ...
A young woman with three kids died within 18 days of being diagnosed with bile duct cancer, which the doctors initially ...
Approved, Seladelpar Can Provide an Important Treatment Option for People Living With the Rare Liver Disease in the European Economic Area – ...
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