Can An Orphan Drug Be Approved Without Clinical Trial?
FDA advisory committee will consider Orphan Europe's NDA for Carbaglu in NAGS deficiency, which is based on data from retrospective review of patient experience with carglumic acid
FDA advisory committee will consider Orphan Europe's NDA for Carbaglu in NAGS deficiency, which is based on data from retrospective review of patient experience with carglumic acid