Collaboration is good news for patients of rare disease

William Sly, M.D., Professor in the Department of Biochemistry, will collaborate with Ultragenyx Pharmaceutical Inc. on the development of enzyme replacement therapy (ERT) for Mucopolysaccharidosis (MPS) Type VII patients. MPS VII is an extremely rare genetic metabolic disease, which was first discovered by Dr. Sly in 1973 and is also known as Sly Syndrome. Although ERT is available for many other MPS diseases, no enzyme therapy has yet been developed for MPS VII due to the small number of patients.

Ultragenyx has in-licensed the enzyme treatment developed by Dr. Sly’s research group in the hopes it can be made available to MPS VII patients. Please see the press release from Ultragenyx for more information: Ultragenyx Press Release.

Department of Biochemistry and Molecular Biology