Ultragenyx Pharmaceuticals Receives Approval of Clinical Trial Application (CTA) for a Phase 1/2 Trial Testing UX003 in Mucopolysaccharidosis Type 7 (MPS 7)
8/14/2013 8:03:36 AM
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NOVATO, Calif., Aug. 14, 2013 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc., a biotechnology company, received Clinical Trial Application (CTA) approval from the Medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K. to conduct a Phase 1/2 clinical trial of UX003, recombinant human ß-glucuronidase, in MPS 7, a rare autosomal recessive lysosomal storage disorder characterized by a deficiency of the enzyme ß-glucuronidase. MPS 7 is a severe multi-system disease resulting in cellular and organ dysfunction. There is no approved drug therapy.
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