Following a fruitful meeting with the FDA, Ultragenyx (RARE) plans to seek accelerated approval for UX111 for the treatment of Sanfilippo syndrome type A.
Ultragenyx's (RARE) pipeline candidate setrusumab shows sustained reductions in fracture rates in patients with osteogenesis imperfecta in new data from the Orbit study.
Ultragenyx (RARE) reports meeting primary and secondary endpoints in the late-stage study of its investigational gene therapy, DTX401, to treat glycogen storage disease type Ia.