First Treatment of Sly Syndrome Patient

Sly infusion 2013

MepseviiTM, the first treatment for MPS VII (mucopolysaccharidosis VII) or Sly syndrome, is now approved by the FDA and in Europe. The treatment was developed by Ultragenyx, a company dedicated to finding therapies for rare and ultra-rare diseases. Dr. William Sly, Emeritus Chair and Professor of Biochemistry, described the first patient with MPS VII, and also conducted research throughout his career based on finding a treatment for this very rare disease. The results of his research on enzyme replacement therapy led to the development of this treatment.

MPS VII is a rare genetic metabolic lysosomal storage disorder caused by deficiency of beta-glucuronidase, which results in a build-up of glycosaminoglycans in tissues and organs. Mepsevii is a recombinant form of beta-glucuronidase designed to treat non-neurological symptoms of the disease.

Department of Biochemistry and Molecular Biology