n-Lorem Foundation announced a new partnership with Ultragenyx Pharmaceutical, a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases.
Novartis picked up a priority review voucher in a $130 million deal with California-based Ultragenyx Pharmaceutical Inc.The PRV is for rare pediatric diseases.
Ultragenyx has revealed results from a Phase 3 trial into the treatment of GNE myopathy showed that its compound did not successfully meet the primary or secondary endpoints of the study.