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Ultragenyx Pharmaceuticals Investigational New Drug Application for UX007 is Active


8/26/2013 9:21:56 AM

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NOVATO, Calif., Aug. 26, 2013 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. announced today that its Investigational New Drug (IND) application filed for UX007 is now active. UX007 is a substrate replacement therapy intended for use in treatment of long-chain fatty acid oxidation disorders (LC-FAOD). It is designed to replace deficient substrates in the fatty acid oxidation pathway and in the tricarboxylic acid (TCA) cycle, the key energy-generating processes in LC-FAOD patients. The active IND allows the company to proceed with its clinical development program for UX007 in LC-FAOD in the US.

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