Studying and finding treatments for rare diseases such as hemophilia and cystic fibrosis require hundreds of thousands of dollars in funding, as researchers at the Penn Center for Orphan Disease Research and Therapy in the Perelman School of Medicine know all too well.
Orphan diseases—which total more than 7,000—represent a collection of disorders that afflict fewer than 200,000 individuals for any single disease type. Approximately 25 million Americans suffer from orphan diseases, but research in most disease types has lagged far behind other major areas.
To help their cause and raise funds for research, the Center, along with the group Rare Disease Cycling, is holding its first annual “Million Dollar Bike Ride” through Philadelphia on Saturday, May 3.
“The goal is to raise money and to provide grants for research on [orphan] diseases, raise awareness about the Center, and increase faculty participation,” says Monique Molloy, one of the coordinators of the ride and a project manager in the Penn Gene Therapy Program.
Individual cyclists as well as teams can register. The ride begins and ends at Highline Park at 31st and Chestnut streets. Bikers can choose three different routes covering three separate distances: 11 miles, 35 miles, or 73 miles.
Cycling teams have been formed to raise money for studying and treating a specific orphan or rare disease. “Bike to End Duchenne” is raising funds to battle Duchenne muscular dystrophy, a degenerative disease that can lead to muscle weakness in areas such as the heart and diaphragm, which ultimately limits life expectancy.
Funds raised by the bicyclists for a specific rare disease will be matched by private and corporate donations. Money raised by registrations, pledges, direct contributions, and matching funds will be directed to a pilot grant focused on the sponsoring foundation’s rare disease.
For more information, visit the Million Dollar Bike Ride website.
Penn Medicine launched the first-of-its-kind, interdisciplinary Center for Orphan Disease Research and Therapy in 2011 with a $10 million gift from an anonymous donor. Its mission is to facilitate and expedite the development of novel therapies for orphan disorders and diseases. H. Lee Sweeney, the William Maul Measey Professor of Physiology, is the Center’s director.
Originally published on March 6, 2014