Orphan Disease Center


Scientific Symposia

The Orphan Disease Center will facilitate several scientific symposia throughout the year focusing on varying areas of rare disease research and therapy. For an opportunity to co-host a symposium on your rare disease focus, please submit a proposal through the link below.

Click here to submit a proposal.

DMD Symposium

On Thursday, April 2nd and Friday, April 3rd, Dr. Jim Wilson and the Orphan Disease Center hosted a scientific symposium on Duchenne Muscular Dystrophy.  Leading scientists, researchers, and industry members in the DMD field participated in this two-day conference at Penn to share pre-clinical data, discuss clinical issues, immunology, AAV manufacturing, and the future of gene therapy for DMD. 


Drug Discovery for Nonsense Mutations in Cystic Fibrosis

On Wednesday, January 21st, 2015, Dr. Kevin Foskett, the Isaac Ott Professor and Chair of the Department of Physiology in the Perelman School of Medicine at the University of Pennsylvania, in partnership with The Orphan Disease Center and Emily's Entourage, hosted a scientific symposium to accelerate research for nonsense mutations of Cystic Fibrosis. Leading CF experts from industry, academia and the Cystic Fibrosis Foundation joined together to collaborate, share research and ideas, and establish an agenda for the development of new treatments for nonsense CF mutations.

Participants enjoyed a cocktail reception and dinner in advance of the symposium at The White Dog Cafe.

Cystic Fibrosis Symposium with Emily's Entourage

For more information about Emily's Entourage, see https://emilysentourage.org

Million Dollar Bike Ride

The second annual Million Dollar Bike Ride, held on Saturday, May 9th, 2015, was a huge success! The Orphan Disease Center welcomed over 500 cyclists and more than 100 family members and volunteers from 29 different US states, plus Canada, the UK, Chile, and the Netherlands. The 2015 MDBR included 23 different teams representing 20 different rare diseases. Now after two years, the MDBR has raised more than $2 million for rare disease research! We look forward to the next ride – date to be announced soon!

Click here for more information.