Ultragenyx's 'Fall From Grace' — Shares Crash 44%, Hitting A Record Low The pharmaceutical industry has long been accustomed to high stakes failures, but when it comes to treating rare genetic disorders like Angelman syndrome, the consequences can be devastating.
Last week's crash of Ultragenyx Pharmaceuticals' stock, down 44% and hitting an all time low, serves as a stark reminder that even with cutting edge science, seemingly promising leads are often fraught with uncertainty.
The failure of apazunersen, a treatment aimed at addressing developmental delays, balance problems, and speech impairment associated with Angelman syndrome, was unequivocal.