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Highlights from the U.S. Conference on Rare Diseases and Orphan Products

Highlights from the U.S. Conference on Rare Diseases and Orphan Products

Role of Academic Centers, Looming Investment Crisis and More.

Jonathan Leff, MBA, Managing Director, Warburg Pincus, spoke in a special session on "Facing the Crisis in Biomedical Innovation: A Venture Investor's Perspective" on Day 2 of the conference.

Duke University Professor of Pediatrics Priya Kishnani, MD, told a standing-room-only audience the intensely moving story of the development of a treatment for infants and children with Pompe disease.  She focused on collaboration among academic researchers, industry and patient advocacy groups, describing the roles of Duke and Genzyme in developing an enzyme replacement therapy.

Lisa Coles, PhD, of the University of Minnesota's Center for Orphan Drug Research, described the expanding role of academic centers in drug discovery and development for rare disease R & D.  She presented strategies related to discovery of new chemical entities and drug repurposing.

FDA representatives and patient advocates discussed the changing role of patients at FDA and the agency's increased emphasis on interaction with patients.

On the Research/Regulatory track, sessions focused on endpoint development and post-marketing period opportunities for continue learning, among other things.  

The APF is active in the Rare Disease Community.

"Remember....Research is the key to your cure!"


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