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.