Press Announcements > FDA approves treatment for rare genetic enzyme disorder
FDA approves treatment for rare genetic enzyme disorder
The U.S. Food and Drug Administration today approved Mepsevii (vestronidase alfa-vjbk) to treat pediatric and adult patients with an inherited metabolic condition called mucopolysaccharidosis type VII (MPS VII), also known as Sly syndrome. MPS VII is an extremely rare, progressive condition that affects most tissues and organs. Continue reading.