Ultragenyx Advances Clinical Development of UX003 for the Treatment of Mucopolysaccharidosis Type 7 (MPS 7)

News | 05. 15. 2013

Ultragenyx Pharmaceutical

 
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.