We're Fundraising to #CureFDMAS
Dear Friends and Family!
On June 8th we're heading back to Philly to ride our bikes in UPenn's Million Dollar Bike Ride that supports rare diseases including ours. This year our team of Wahoo Riders has nine riders ranging in age from nearly 10 to 81!! Together we're raising funds to support critical research to find a cure for Fibrous Dysplasia/McCune Albright Syndrome. As you know we have been intimate with FD ever since Liam was diagnosed at age four and has since endured several painful hip and femur surgeries and long recoveries. Fortunately the kid, now 16, is unstoppable and finds great enjoyment in fishing, boating, snowboarding and surfing. Still, regular bone pain and discomfort is a reality for Liam which motivates us to do what we can to help researchers move this disease toward a cure. We're doing it for Liam and others who have FD/MAS, and for children who will be born with it.
From Liam: "Hi Everyone, thank you so much for donating last time. With your help we raised more money than we thought possible, $13,746! We're committed to doing this ride again and this year we have a big group of family and friends biking with us so all your support will help us move forward to finding a cure."
FD/MAS affects one in a million people, which is indeed rare until someone you love has it. While we can joke sometimes and even Liam will say, "Yep, I'm one in a million!" we've learned along the way that rare diseases get significantly less attention and funding. So we're getting louder and by partnering with the University of Pennsylvania's Million Dollar Bike Ride our collective action is making impactful progress. This event brings together a large sum of people either living with or supporting someone living with a rare disease. Please know that 100% the funds we Wahoo Riders generate along with the other FD/MAS teams will go DIRECTLY to cutting-edge research grants. UPenn also generously matches our first collective 30K with an additional 30K, Wahoo!
Those of you who joined up for our inaugural fundraiser last year will be pleased to know we far exceeded our personal expectations and the collective FD/MAS teams raised a new record. We aim to top that this year. You can see below how your money was used in 2018.
If you are able to join our mission again this year we will be honored to carry your support with us to Philly and beyond.
With love and appreciation,
Kim, Brian, Liam, Dessa, Sorelle, Paprika, Jeni, Sam and John
This Bike Ride POWERS Research
100% of the donations to this fundraiser will go DIRECTLY to cutting edge research grants, with the Penn Orphan Disease Center matching the money we raise up to an additional $30,000. In our first four years with the Million Dollar Bike Ride, we've raised over $570,000 for FD/MAS research. Funded studies go through a competitive review process.
This Bike Ride is our search for a cure. It is the most direct way for the FD/MAS community to ask the most pressing scientific questions and fund the most promising therapuetic possibilities for a treatment.
Here's where funding went:
- $68,178 to Identification and Characterization of Novel Cell-Permeable, Small Molecule Adenylyl Cyclase Inhibitors for Future Development as Drugs to Treat FD/MAS, Dr. Charles Hoffman, Boston College
- $68,178 to Single Cell Transcriptome Analysis of Skeletal Stem Cells Derived from FD/MAS Patients, Dr. Fernando Fierro, University of California Davis.
- $68,178 to Elucidating the Role of GNAS Mosaicism in Fibrous Dysplastic Lesions, Dr. Kelly Wentworth, University of California, San Francisco
- $68,178 Anti-resorptive drugs in fibrous dysplasia of bone: Studies on the effects of a RANKL inhibitor and Zoledronic Acid in a murine model of the disease by radiography, histology, and genome-wide expression analysis (NanoString), Dr. Mara Riminucci, Sapienza University of Rome. Learn more here.
- $53,614 to Dr. Mara Riminucci of Sapienza University of Rome for her project: “Exploring the therapeutic potential of RANKL inhibition in Fibrous Dysplasia of bone: studies on murine transgenic models of the disease,” and
- $53,614 to Dr. Yingzi Yang of Harvard School of Dental Medicine for her project: “Mechanistic and Therapeutic Studies of Fibrous Dysplasia in a New Mouse Model.”
- How do these studies fit in to the fight to #CureFDMAS?: Learn more here.
- $58,500 to Dr. J. Silvio Gutkind of Development of Moores Cancer Center, UC San Diego for his project: "Next-Gen Animal Models for Fibrous Dysplasia(FD)/McCune-Albright Syndrome(MAS)"
- $58,500 to Dr. Mara Riminucci of Sapienza University of Rome for her project: "Mouse Models of Fibrous Dysplasia as a Tool for Developing Rational and Effective Therapies."
- How do these studies fit in to the fight to #CureFDMAS?: Learn more here or from this update.
Want more information on Fibrous Dysplasia/McCune-Albright syndrome?
FD/MAS is a rare and debilitating disease that has no FDA-approved treatment. It causes bone tumors to grow in place of normal bone, and often causes chronic pain, loss of mobility, hormonal imbalances, skin marks, deformity and more. There's no cure, and no way to slow it down. Bone-forming cells fail to mature and areas of healthy bone are replaced with this fibrous tissue. The severity of the disease covers a wide spectrum. It can affect a single bone and go unnoticed for years or can affect multiple bones or virtually every bone, start very early in life, and result in significant physical impairment, deformity and severe bone pain. FD can also be associated with birth marks (cafe-au-lait spots), and a number of endocrine problems such as precocious puberty, hyperthyroidism, low blood phosphorus, and excess growth hormone. When this occurs, this is known as McCune-Albright syndrome (MAS). (Liam deals with the FD part but doesn't have issues associated with McCune-Albright Syndrome.) The cause of the gene defect in FD/MAS is unknown, but the defect is neither inherited from the person's parents, nor passed on to the person's children. When the long bones (the bones of the legs and arms) or flat bones (ribs and pelvis) are affected, the bones weaken, may bow, are often extremely painful, and will frequently fracture. Affected bones in the skull often expand,cause facial disfigurement and, again, can be very painful both physically and emotionally.
For More Details and to Register to Ride
To learn more about the Million Dollar Bike ride, the research grant process, and how you can participate, Check out our page at https://secure.qgiv.com/event/2019mdbr/