Abandoned, Shelved, and Rescued Therapies for Ultra-Rare Diseases

Drug development programs for ultra-rare diseases may be discontinued for reasons unrelated to scientific validity, including resource constraints or shifting priorities. Presenters will describe case studies of therapies that were abandoned for commercial or logistical reasons and later revived through academic leadership, public-benefit models, or through repositioning.

Moderator: Edward Neilan, MD, PhD, Chief Medical and Scientific Officer, NORD

Panelists:

• Paul Ayoub, PhD, MBA, Founder and CEO, Rarity PBC
• Terry Pirovolakis, Founder & CEO, Elpida Therapeutics 
• Matthew Porteus, MD, PhD, Professor of Pediatrics, Stanford University, a NORD Rare Disease Center of Excellence

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