Financial help available for MPS VII patients

The Assistance Fund, an independent charitable patient assistance foundation that helps patients and families facing high medical out-of-pocket costs, has launched a financial support initiative for MPS VII patients and … Continued

FDA approves MPS VII drug

The U.S. Food and Drug Administration today approved Mepsevii (vestronidase alfa-vjbk) to treat pediatric and adult patients with MPS VII, Sly syndrome. The safety and efficacy of Mepsevii, developed by … Continued

Gene Therapy for MPS IVA

Mucopolysaccharidosis IVA (MPS IVA: Morquio A syndrome) is caused by a deficiency of N-acetylgalactosamine-6-sulfate sulfatase. Deficiency of this enzyme progressively causes accumulation of the glycosaminoglycans keratan sulfate and chondroitine-6-sulfate in … Continued