NasdaqGS:RAREBiotechs
Ultragenyx Pharmaceutical (RARE) Is Down 31.0% After Rare-Disease Milestone Updates - Has The Bull Case Changed?
Ultragenyx Pharmaceutical recently reported two key milestones: strong Phase 3 results for setrusumab (UX143) in Osteogenesis Imperfecta and completion of its Biologics License Application to the FDA for DTX401 gene therapy in Glycogen Storage Disease Type Ia, supported by up to six years of clinical follow-up data.
These advances highlight Ultragenyx’s push to convert late-stage rare disease programs into potential commercial therapies that could broaden its treatment portfolio and...