By SPC News Staff
The FDA approved vestronidase alfa-vjbk (Mepsevii, Ultragenyx Pharmaceutical) to treat pediatric and adult patients with mucopolysaccharidosis type VII (MPS VII), also known as Sly syndrome.
An inherited metabolic lysosomal storage disorder, MPS VII is a rare, progressive condition that affects most tissues and organs. MPS VII is one of the rarest MPS disorders, with an estimated 200 patients in the developed world. The features of MPS VII vary widely from patient to