FDA Regulatory Flexibility Under Fire as Gene Therapy Holds Stall Rare Disease Progress

By Kat Bryant Knudson · STAT News · 2026-07-31

FDA Regulatory Flexibility Under Fire as Gene Therapy Holds Stall Rare Disease Progress

According to STAT News, the implementation of platform technology designations intended to accelerate drug development is instead creating significant roadblocks for limb girdle muscular dystrophy patients. Despite federal mandates to modernize review processes, the agency has be

According to STAT News, the implementation of platform technology designations intended to accelerate drug development is instead creating significant roadblocks for limb girdle muscular dystrophy patients. Despite federal mandates to modernize review processes, the agency has be