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Ultragenyx Announces Positive Data From Phase 2 Study of Sialic Acid Extended-Release

Ultragenyx Pharmaceutical today announced the presentation of detailed results from a 48-week Phase 2 clinical study of sialic acid extended-release (SA-ER, UX001) tablets in 47 patients with hereditary inclusion body myopathy (HIBM; also known by its new name as GNE myopathy), a rare, progressive muscle-wasting disease.