Parents of children and young adults with the rare disease Congenital Muscular Dystrophy (CMD), Susan Lee-Miller, Andy Parks, and Diane Smith-Hoban, are spending the next seven weeks preparing for the 5th Annual Million Dollar Bike Ride (MDBR), to be held in Philadelphia on May 20th.
The bike ride, hosted by the Penn Medicine Orphan Disease Center (ODC), is a 13-, 34-, or 72-mile route through the Greater Philadelphia region, and has raised more than $6.4 million for rare disease research since its inception in 2014. Lee-Miller, Parks, and Smith-Hoban each have a child affected by Collagen-VI Congenital Muscular Dystrophy (CMD), so they are riding with Team Cure CMD. Their goal is to raise $100,000 for COL6 CMD. To ride with Team Cure CMD and/or to donate, visit http://www.tinyurl.com/RideWithCureCMD.
Each rare disease team, including Team Cure CMD, must raise at least $20,000 to earn ODC matching dollars up to $50,000. The ODC ensures that all dollars raised go directly to the pilot grants, with no overhead costs. This unique opportunity to fundraise helps build awareness for causes that many people have never heard of. There is no limit to the size of a team, and Team Cure CMD members can be “virtual,” meaning they do not have to be present on ride day to join the fundraising effort.
On the day of the ride, Team Cure CMD cyclists, affected individuals, and their families and supporters will gather at 31st St. and Chestnut near the University of Pennsylvania campus. While cyclists complete their routes, non-riders can enjoy food and drinks donated by local vendors, indoor putt-putt and other games, video displays, and community-building. Rare disease team members connect, share information, and become friends. Parks’ wife, Jessie, says “The strength, inspiration, and hope that comes along with being around other people in the rare disease community is priceless.”
Smith-Hoban from Lafayette Hill, PA, rides with family and friends and says that the 13-mile ride is beautiful and relatively easy. “This route goes through Fairmount Park and shows off iconic Philadelphia scenes such as Boathouse Row and other well-known sites.”
Lee-Miller, hailing from Doylestown, PA, rides the tougher 34-mile while her husband, Scott completes the intense 72-mile trek. Their 20-year-old son Liam, who is in his second year at Temple University, hangs out with Smith-Hoban’s son, Luke, a 22-year-old recent Penn graduate. The two young men both live with CMD and have been good friends and wheelchair hockey teammates for more than 10 years.
Parks from Fairfax, VA, also rides the 72 miles through the greater Philadelphia area. His wife Jessie brings their young daughters Naya and Divia to provide moral support for the team. This will be their second year participating in the event, which they learned about when they saw a post in a CMD support group they had joined when Divia was first diagnosed. “We have been learning a lot. With each event we get ourselves involved in, the disease doesn’t feel as intimidating and we are not as helpless in our fight against it.”
Cure CMD was founded in 2008 to advance research for treatments and a cure for congenital muscular dystrophy (CMD and, through engagement and support of the CMD community, to also improve the lives of those living with CMD. To date, the organization has funded more than $2 million in research, helped launch effective animal models, and has connected more than 2,000 affected individuals to a supportive, helpful community. Visit www.curecmd.org for more information.
The Penn Medicine Orphan Disease Center (ODC)
The Penn Medicine Orphan Disease Center (ODC) was established to promote the development of therapies across a broad range of orphan diseases. Each of these diseases by definition is rare but collectively represents a substantial health care burden. Most orphan diseases manifest in children with premature mortality and/or significant disability and little is available in terms of treatments. In order to assemble a critical mass of intellectual and patient resources for any one disease, the ODC will reach beyond the borders of Penn and promote inter‐institutional collaborations/partnerships. The ODC will develop transformative therapies using platform technologies that can be deployed across multiple rare diseases. We will emphasize disorders with substantial unmet need independent of their incidence and will strive to assure access to patients of all populations.
All funds raised by Disease Teams through pledges are used by the ODC to expand the scope and depth of the MDBR pilot grant program. Following the event, a Request for Application (RFA) will be broadly distributed to the international scientific community requesting pilot grant applications to study diseases for which designated money has been raised. The ODC secures philanthropic and corporate donations that will be used to match dollar‐for‐dollar money raised by the cycling participants. Scientific leadership of the ODC and its extramural advisors will review the applications and make awards to those of the highest scientific merit which address the specific topics outlined in the RFA. The ODC will disperse the money to grantees and manage progress of the science and spending on the award.
For questions regarding participation in the MDBR as a cyclist, volunteer or sponsor, or regarding the pilot grant program, please contact Samantha Charleston at firstname.lastname@example.org or (215) 573-6822.