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Ultragenyx Pharmaceutical Inc.

Drug Names(s): UX001, UX-001 sialic acid extended release (SA-ER), aceneuramic acid extended release

Description: UX001 is an extended release formulation of sialic acid that should allow themaintenance of steady levels of sialic acid after oral administration. The sialic acidreplacement therapy is expected to restore the proper biochemistry of glycoproteinsand glycolipids by allowing proper sialylation. Data from an HIBM (Hereditary Inclusion Body Myositis) mouse model show aprofound beneficial effect of sialic acid replacement therapy on the biochemistry,pathology and clinical outcomes of HIBM mice.

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