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 on the placebo-controlled, Phase 3 RESTORE study in 13 adult ...
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 on the placebo-controlled, Phase 3 RESTORE study in 13 adult ...
