Orphan Disease Center


Million Dollar Bike Ride

The third annual Million Dollar Bike Ride will be held on Saturday, May 7th, 2016 at Highline Park (31st & Chestnut Streets). Registration opens this fall. The ride includes a 12, 33, or 73 mile option, and plenty of refreshments and entertainment for post-ride.  Whether you're an avid or beginner cyclist, we hope you'll join us to raise awareness and much-needed funds for rare disease research! For questions, contact Samantha Charleston at scharle@upenn.edu or (215) 573-6822.

Click here for more information.

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.

Congenital Hypoglycemia Disorders: Hyperinsulinism and GSD

Date: April 14-April 15, 2016, 8:00 am - 5:00 pm

Course Overview: This two-day international symposium will be an update on recent exciting advances in research related to congenital hyperinsulinism and other inherited hypoglycemia disorders, particularly disorders of glycogenoses.  The symposium is intended to enhance the clinical management skills and practices of endocrinologists, metabolism specialists, neonatologists, surgeons, pathologists, radiologists, geneticists and nurses, as well as basic scientists interesed in pancreatic islet biology and regulation of glucose metabolism.

Click here for more information. Click here for event brochure.

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