News | News By Subject | News by Disease News By Date | Search News
Get Our FREE
Industry eNewsletter
email:    
   

Ultragenyx Initiates Phase 2 Study of UX001 in Hereditary Inclusion Body Myopathy, a Rare Neuromuscular Disease



7/5/2012 9:24:47 AM

NOVATO, Calif., July 5, 2012 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced the dosing of the first two patients in a Phase 2 study of UX001 for hereditary inclusion body myopathy (HIBM). HIBM is a rare, severe, neuromuscular disease caused by sialic acid deficiency. UX001 is an extended-release oral tablet formulation of sialic acid (SA-ER) intended as a substrate replacement therapy for HIBM.

Read at GlobeNewswire


comments powered by Disqus
   

ADD TO DEL.ICIO.US    ADD TO DIGG    ADD TO FURL    ADD TO STUMBLEUPON    ADD TO TECHNORATI FAVORITES