For the 95% of rare disease patients still without an approved treatment, FDA is working for you. Acting Commissioner Kyle Diamantas shares how FDA is transforming rare disease drug development through the new Rare Disease Framework Initiative. Because every family deserves answers, action, and hope. fda.gov/news-events/fda-voic…
Delivering Answers and Hope for Rare Disease Families

Delivering Answers and Hope for Rare Disease Families

Rare diseases present our most formidable challenge and our greatest opportunity to speed life-saving treatments and cures to the patients who need them most.

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Non-ambulent Duchenne Muscular Dystrophy boys have NOTHING to help them preserve upper body function. Until now. DERAMIOCEL. The FDA has the data - and it WORKS, obviously - 76% reduction in loss of upper limb function. That's MEANINGFUL - please approve it - STAT - every day they lose more muscle!

Nov 5, 2026 · 6:52 PM UTC

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