Photos from the 2014 Rare Disease Forum at Whitehead Institute

On Thursday, February 27th Whitehead Institute hosted its 3rd annual Rare Disease Forum in recognition of Rare Disease Day.  This year’s focus was on “Curing Rare Childhood Diseases: New Industry and Academic Models” and attendees heard from leaders in rare and orphan disease research in the Boston area.  Featured speakers included Alan Beggs, Director of The Manton Center for Orphan Disease Research, Jeffrey Leiden, President and CEO, Vertex Pharmaceuticals, David Meeker, President and CEO, Genzyme, and David Williams, Chief, Division of Hematology/Oncology and Director of Translational Research, Boston Children’s Hospital. Whitehead Member Harvey Lodish moderated the panel portion of the evening where advocacy groups were invited to ask questions.  Rare disease patient advocacy organizations included the Association for Frontotemporal Degeneration, the Caring for Carcinoid Foundation, the Hermansky-Pudlak Syndrome Network, the National Tay-Sachs and Allied Diseases Association, the Pitt Hopkins Research Foundation, and the VHL Alliance.

Following the discussion, Whitehead hosted a networking session where attendees had the opportunity to discuss the various challenges and opportunities faced in the advocacy and promotion of rare disease research. Below are photos from the event.

Whitehead Member Harvey Lodish moderated the discussion with David Williams, David Meeker, Alan Beggs and Jeffrey Leiden.

David Williams gave an overview of the important collaboration between Boston Children’s Hospital and a local biotech in the development of rare disease therapies.

Jeffrey Leiden explained the story of the invaluable partnership between Vertex and the CF Foundation in the development of the Cystic Fibrosis drug therapy.