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Ultragenyx Pharmaceuticals Release: Sialic Acid Extended-Release Phase 2 Study Data To Be Presented At American Academy of Neurology Annual Meeting


4/28/2014 9:05:29 AM

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NOVATO, Calif., April 28, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the upcoming presentation of data from a Phase 2 clinical study of sialic acid extended-release (SA-ER, UX001) tablets in hereditary inclusion body myopathy (HIBM; also known by its new name as GNE myopathy). HIBM is a rare, progressive muscle-wasting disease, and SA-ER is designed to replace the deficient sialic acid substrate in patients with HIBM.

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