FDA Approves Ultragenyx Enzyme Replacement Therapy for MPS VII

Ultragenyx Pharmaceutical has won FDA approval for its enzyme replacement therapy Mepsevii™ (vestronidase alfa), the first treatment authorized by the agency for children and adults with mucopolysaccharidosis VII (MPS VII).Mepsevii is an enzyme-replacement therapy designed to replace the deficient lysosomal enzyme beta-glucuronidase in patients with MPS VII, a rare genetic metabolic lysosomal storage disorder (LSD) caused by the deficiency of beta-glucuronidase.MPS VII—which accordin