Ultragenyx Pharmaceuticals Announces a Positive Signal in Interim Data from Phase 2 Study of UX001 in Hereditary Inclusion Body Myopathy
7/3/2013 7:48:13 AM
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NOVATO, Calif., July 3, 2013 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, announced interim 24-week data from a 48-week Phase 2 clinical study of UX001 in 47 patients with hereditary inclusion body myopathy (HIBM), a progressive muscle-wasting disease. The study compared treatment with a total daily dose of 6 grams or 3 grams of UX001 with placebo. UX001, an oral sialic acid extended-release (SA-ER) tablet, is designed to replace the deficient sialic acid substrate in patients with HIBM.
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