-
A Breakthrough for GSDIa: FDA Grants Accelerated Approval to Vyjuvek’s Counterpart, Lonapegsomatropin (Egtilparvovec)
NASDAQ: $RARE A Milestone for Glycogen Storage Disease Type Ia The landscape of rare disease treatment has reached a significant milestone. The U.S. Food and Drug Administration (FDA) has officially granted accelerated approval to Lonapegsomatropin (Egtilparvovec), marketed as GenGlymyl (pariglasgene brecaparvovec-opnr), a pioneering gene therapy developed for the treatment of adults with Glycogen Storage Disease…