Ultragenyx Pharmaceutical

Ultragenyx initiates phase I UX001 trial

Tuesday, August 16, 2011 01:52 PM

Ultragenyx Pharmaceutical, a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, has begun the dosing of the first patient in a phase I study of UX001 for hereditary inclusion body myopathy (HIBM). UX001 is an extended release formulation of sialic acid intended as a substrate replacement therapy for HIBM, a severe, progressive, genetic neuromuscular disease caused by sialic acid deficiency. UX001 is the first program from the company's pipeline to enter the clinic since its founding in 2010.

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