Dossier
rare disease community
Coverage of rare disease community in the Nexus archive.
- My son has a terminal disease — why FDA delays are failing families like mine
A mother caring for her son with Duchenne muscular dystrophy criticizes the FDA for regulatory delays in approving experimental drugs, arguing that bureaucratic caution harms terminally ill children. She calls for FDA Commissioner Dr. Martin Makary to address the issue and appoint a successor to Dr. Vinay Prasad who prioritizes patient access over caution.