Closing the Rare Disease Innovation Gap October 07, 2016 Share this page The Food and Drug Administration recently approved a novel therapy for Duchenne muscular dystrophy utilizing this special pathway when the science is imperfect but the disease is deadly. We support the approval but also note that the development program left many questions unresolved Related Articles FDA Rare Disease Roundtable – June 3, 2026 June 04, 2026 Rare Disease Week 2026: Senate Hearing Recap February 27, 2026 FDA's Rare Disease Innovation Hub Meeting: Key Takeaways October 22, 2024 See All News