pharmaphorum8h
FDA clears first therapy for rare disease CTX from Mirum
Mirum Pharma's Ctexli has become the first FDA-approved therapy for cerebrotendinous xanthomatosis (CTX), an ultra-rare form ...
Mirum’s CTEXLI™ (chenodiol) Tablets Receives FDA Approval for Treatment of Cerebrotendinous Xanthomatosis (CTX)
(Nasdaq: MIRM) today announced that the U.S. Food and Drug Administration (FDA) has approved CTEXLIâ„¢ (chenodiol) tablets, a ...
US FDA approves Mirum Pharma's genetic disorder drug
The U.S. Food and Drug Administration approved Mirum Pharmaceuticals' genetic disorder drug on Friday, making it the first ...
MedPage Today2d
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 indicated for the rare lipid storage disease and autosomal recessive genetic ...
Monthly Prescribing Reference3d
FDA Approves Ctexli for Cerebrotendinous Xanthomatosis
Ctexli replaces deficient levels of the endogenous bile acid chenodeoxycholic acid, reducing the abnormal deposits of cholesterol metabolites that have been associated with organ and tissue damage ...
Nature2y
BILIARY BILE ACID EXCRETION BY THE HUMAN FETUS DURING EARLY GESTATION
Chenodeoxycholic and cholic acids were the major bile acids identified however the profiles were characterized by an array of metabolites not normally found in adult bile. Hyocholic acid levels ...