I'm Fundraising to #CureFDMAS
Our amazing daughter Carly has been bravely battling Fibrous Dysplasia (FD) in her hip and femur for the past 11 years. At 13, Carly was an active teenager and competition dancer when she was diagnosed with this rare and incurable bone disease after fracturing her hip. Since that time, Carly has endured five painful surgeries and grueling medical treatments. Despite living with pain and limitations, Carly is an inspiration to everyone she encounters with her strength and determination. She dedicates herself to helping others with FD as an active fundraiser for the FD Foundation and as an administrator of a Facebook support group. Carly is also part of a team that created an FD patient registry. Please help Carly and the thousands of children and adults afflicted with FD. Many of them suffer with FD lesions (benign tumors) in various parts of their bodies, including their faces and skulls, causing debilitating pain and disfigurement. In addition to disease of their bones, other FD patients also have may suffer from a wide range of endocrine issues and cafe-au-lait skin spots (when this occurs it is called McCune Albright Syndrome (MAS)).. Your contribution to our team will bring us one step closer to beating this terrible disease. Every dollar raised is going to a DIRECT research grant for a treatment or cure for fibrous dysplasia, and the Penn Orphan Disease Center will be matching the money we raise up to an additional $30,000.
Please help the Team FD be the #1 rare disease team for 2019 and #CureFDMAS! Thank you for your support.
The Levin Family, Captains of Team FD
To see our Full Team FD page with all of the fundraisers, click here.
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). 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.
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.
For More Details and to Register to Ride
To learn more about the Million Dollar Bike Ride being held on June 8th, the research grant process, and how you can participate, Check out our page at https://secure.qgiv.com/event/2019mdbr/