MPS VII Archives - MPS Society

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

Therapeutic Targeting of Wnt/β-Catenin Signaling to Improve Bone Formation in MPS VII

Skeletal disease is prevalent in most mucopolysaccharidosis (MPS) subtypes, particularly in the spine, where vertebral dysplasia leads to progressive def01mity and spinal cord compression. Resulting pain and paralysis significantly diminish … 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