
Ep. 188 – Pharma deals; Plus: Genentech, Leerink Conversations
BioCentury This Week
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The Curious Case of Stealth Therapeutics
A company is trying to develop a drug for an extraordinarily rare disease called Barth syndrome. It affects about 150 Americans and 85% of patients die before the age of five. They've been struggling to get it approved at FDA, getting shifted around from one division to another. The only way they think they can make any money with that drug candidate is if they get a rare pediatric priority review voucher.
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