Ultragenyx Advances Clinical Development of UX003 for the Treatme...

NOVATO, CA - May 15, 2013 - Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced a Phase 1/2 study of UX003 for mucopolysaccharidosis type 7 (MPS 7, or Sly Syndrome). UX003 is a recombinant human β-glucuronidase intended as an enzyme replacement therapy (ERT) for the treatment of MPS 7, an extremely rare autosomal recessive lysosomal storage disorder characterized by a deficiency of the lysosomal enzyme β-glucuronidase and a severe multi-system disease. MPS 7 has no approved therapies.

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