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Cycling for Rare Diseases

MDBR full Graphic with Penn logo no full text FinalThe first annual Million Dollar Bike Ride is finally here. On Saturday, May 3, 2014, close to 500 riders and many other volunteers and family members will gather at Highline Park on Penn’s campus to raise funds for and awareness about rare diseases. The Ride is organized by the Center for Orphan Disease Research and Therapy at Penn Medicine and Rare Disease Cycling.

Most of the teams are family members and friends of children with a rare disease -- older brothers held dress-down days at their high schools to raise money, baby-sitters have volunteered to ride, and moms and dads have trained to cycle the 75-mile route in hopes of ultimately finding better treatments for their children. One rider from North Carolina, who is living with Pitt Hopkins syndrome, will celebrate her 16th birthday on the day of the Ride, after training for the last three months with her mom to ride the 11-mile route. There is one entire team of Penn scientists. Another Penn researcher is riding for the team dedicated to familial hypercholesterolemia, his life’s work.

A Penn vet investigator who studies Niemann-Pick Disease Type C (NPC) is riding on this team. This researcher's son and an older brother of a boy who has NPC raised over $6,500 riding their bikes in their respective neighborhoods. The executive director of the ML4 Foundation, based in Atlanta, will be at the Ride as a volunteer. One rider had a brother who died of Tay-Sachs in 1970. And, a young woman from San Diego with the lung disease LAM arrived in Philly yesterday and will be using an oxygen tank to help her during the Ride.

Lee Sweeney, PhD, director of the Penn Center for Orphan Disease Research and Therapy and Jim Wilson, MD, PhD, director of Penn’s Gene Therapy Program and founder of Rare Disease Cycling, will also hit the road.

The Ride is not to benefit just one rare disease, but many. Individual cyclists have registered and raised money for their specific orphan/rare disease foundation, and funds raised to support research for a specific rare disease will be awarded with dollar-for-dollar matching funds, up to a maximum of $50,000.

Orphan/rare diseases represent a collection of more than 7,000 distinct disorders that each afflicts fewer than 200,000 individuals. In all, over 25 million people in the United States are sickened and often die from orphan diseases. Most rare diseases are genetic, and thus are present throughout the person's entire life, even if symptoms do not immediately appear. Many rare diseases start early in life, and about 30 percent of children with rare diseases will die before reaching their fifth birthday. Despite this huge number, research in most disease types has lagged far behind other major areas due to a combination of technological and funding limitations.

The inaugural Ride’s teams are listed here – take a look at each team’s site and get to know the families, the riders, the researchers, and the patients:

Team Familial Hypercholesterolemia
Team Cure ML4 (Mucolipidosis Type IV)
Team ALD (Adrenoleukodystrophy)
Spin Factor for Hemophilia
Bike to End Duchenne (Duchenne Muscular Dystrophy)
LAM Foundation (Lymphangioleiomyomatosis)
Pitt Hopkins Pedalers
Raring to Go for CHI (Congenital Hyperinsulinism International)
RASopathies Network
Team NPC (Niemann Pick Type C)
Team FARA (Friedreich’s Ataxia)
Team NTSAD (Tay-Sachs, Sandhoff and GM1)
Penn Scientists for Orphan Disease Research
Team Cure CMD (Congenital Muscular Dystrophies)
Team MPS (Mucopolysaccharidoses)

Stay tuned for research progress and news of the next Ride in the spring of 2015!


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