Ultragenyx Pharmaceuticals Announces Initiation Of Phase 1/2 Study Of Recombinant Human Beta-Glucuronidase (Rhgus) In Mucopolysaccharidosis Type 7 (MPS 7)
12/4/2013 9:15:02 AM
NOVATO, Calif., Dec. 4, 2013 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc., a biotechnology company, today announced the dosing of the first patient in a Phase 1/2 study of recombinant human beta-glucuronidase (rhGUS, UX003) for MPS 7, in Manchester, UK. MPS 7 is an ultra-rare autosomal recessive lysosomal storage disorder characterized by a deficiency of the enzyme beta-glucuronidase that results in a severe multi-system disease.
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