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All breast cancer trials fertility preservation travel vaccines orphan drug access autoimmune flare management

University Trial Case Study: Global Rare-Disease Wins for Kids

How university-sponsored trials helped families access orphan drugs faster in 2024–2025, with caregiver perspectives, trial logistics, and practical tips for flu season and expanded access.

Robert Maxwell Sep 23, 2025

orphan drug access Pediatric gene therapy Fabry disease trials expanded access programs