This year, we’d like to invite YOU to take action too.
Here’s how we’re doing so far!
Thermometer By ABC Fundraising®
Thermometer By ABC Fundraising®
The FD/MAS Patient Registry is turning 2 on Wednesday, October 31st and we’re celebrating big with Registry Week! Since the Registry launched in 2016, 680 patients and caregivers have signed up to be a part of this study and contributed over 100,000 data points. That’s 100,000 ways that scientists and researchers can learn more about this disease.
Several groups of scientists have proposed projects that will draw on data that Registry participants have shared in the FD/MAS Patient Registry. To conduct their studies, they need more people to complete the surveys, and for everyone who completed their surveys to come back and share how your condition has changed or stayed the same. These Registry studies could lead to better treatment guidelines and a new understanding of what medical procedures make a real difference in patient lives. The work of these researchers is just the beginning; they need YOUR story to power research.
We’re celebrating Registry Week from October 27th to November 5th with new FD/MAS content, and a brand new Registry survey. Help us celebrate by signing in to the Registry and completing and updating your surveys!
While 318 patients have completed at least 1 survey in the Registry, only a portion of these participants have completed all their Registry surveys. When a participant completes all their surveys, scientists are able to see the most full and complete picture of their disease experience. That’s why we’re asking you to celebrate Registry Week with us by visiting the Registry and sharing your story.
Throughout Registry Week, there will be videos, news articles, and a take home prize for anyone who completes all their surveys! Get started on your Registry surveys now and stay tuned for more Registry Week info!
Whether you love the cooler temperatures of fall, or hate it, Aurélie Lagneau of France has given the FD/MAS community a new reason to be enthusiastic about sweater weather. This avid knitter has taken her skill with yarn and her love for her daughter to create a unique fundraiser, Supportive Skein, which has already raised $28,000 for FD/MAS research.
Aurélie and her husband lived a busy life in Paris, France when they welcomed their daughter, Gaïa in 2015. At just one month old, Gaïa was suffering with advanced stages of cushings syndrome, and was diagnosed with McCune-Albright syndrome. Gaïa turned three years old this September, and has already had several surgeries for her FD. “She has had 2 spinal fusions on her neck, so she can no longer move her head,” explains Aurélie, “Since the surgeries, she doesn’t want to eat, so she is fed by gastrostomy.” To focus their attention and take care of their daughter, the Aurélie and her husband left their jobs and moved to the French countryside.
Like many of the patients and families who help the FD/MAS community by fundraising, Aurélie was determined to do something to help her little girl. “Because I am a knitter, I knew how to reach other knitters and designers who might be willing to help,” explained Aurélie. On Rare Disease Day 2018, she launched The Supportive Skein, a fundraiser that taps into the enthusiasm of the knitting community to raise research funds. Through Supportive Skein, crafters can buy knitting patterns and the proceeds all go toward FD/MAS research.
“Every month I had at least a donation from a designer, and around 500€ to 1K€ for FD/MAS research,” said Aurélie, “But quickly, yarn dyers asked to be part of the challenge too. So now I have at least two donations each month. The designers create a pattern, and the dyers create a new colorway and the proceeds of this specific colour skein is donated.” Now, other artists have become interested in helping and Supportive Skeins have brought their work and their fans to the cause. Aurélie now sells mugs, bags, and other items with beautiful artwork and drawings on them as well.
In addition to raising money, Aurélie also works to raise awareness. “From the beginning, I tried to educate people who have no idea about FD/MAS.” On the Supportive Skein Instagram page, Aurélie shares a Wednesday Awareness post with funny pictures and a sentence about the disease or about a FD/MAS patient. She has also helped to launch a new patient organization in France, MASFD has 130 members already and continues to grow.
“In the beginning, my goal was to reach $30,000, but now I plan to reach $60,000 so that we might fund a new research grant entirely,” said Aurélie. She already has plans for developing her own brand of wool dying, and to bring the project from France to reach world wide. “In France, it’s become quite popular. I have many famous designers involved already. Now I’ve reached out to over a dozen designers from across the world to create a calendar of new patterns for 2019.” You can find the beginnings of her website in English here. Aurélie’s goal is to have a full grant ready in 2020 for FD/MAS research.
Aurélie manages the entire project on her own, including recruiting new designers, posting about the new designs and color-ways, and sending out products to customers and lottery winners. It is a clear labor of love for her daughter and an inspiration for how one person’s hobby can become a powerful way to take action. You don’t have to be a PhD fellow. You don’t have to be a wealthy philanthropist. With determination and an idea, Aurélie is taking matters in her own hands and pushing for FD/MAS research.
Visit her store or find her on Instagram here. @echeveausolidaire
One of the exciting aspects of the Million Dollar Bike Ride fundraiser is how quickly the gifts from patients, friends and family go directly towards actual FD/MAS science. In July, UPenn announced that $273,000 would be awarded to the best proposals for FD/MAS studies, and researchers from around the world have already turned in their proposals for the four grants available.
Grants awarded ten months ago in December 2017 are now in the final months of their project period. Dr. Mara Riminucci has continued her work, funded by previous Million Dollar Bike Ride grants, on RANKL inhibitors, a project that looks at FD bone and tries to prevent bone fragility and deformity. Previous Million Dollar Bike Ride grants have helped Dr. Riminucci progress this project forward and test a treatment that causes, in mice, the replacement of fibrotic tissue with bone, while halting the progression of FD, but “what is not properly defined today is the approach that will prevent or reduce the relapse of the disease following treatment withdrawal,” explains Dr. Riminucci. Right now, her lab continues to work with FD mouse models to focus on the exact dosage and course for her treatment so that, eventually, this same therapeutic strategy can help treat patients.
Our other 2017 awardee, Dr. Yingzi Yang is also working on therapeutic strategies that will improve the lives of FD/MAS patients further down the line. Like Dr. Riminucci, Dr. Yang is using mouse models to better understand how FD works on a molecular and cellular level. By learning more about the “underlying mechanisms of FD, we can then identify and test therapeutic targets for FD/MAS,” says Dr. Yang.
Mouse models are a common early step for new drug therapies because they allow researchers to make sure their findings are safe and effective before developing them for patients. This is an important step on the path towards a treatment patients can access in stores. “We hope that we can test some of these therapeutic targets using patient-derived cells,” Dr. Yang explains, highlighting other steps on the path to new treatments “we also hope that, eventually, we can recruit patients in possible clinical trials for these therapies.” Like Dr. Yang’s project, Dr. Riminucci’s project aims to create a treatment that will help patients down the line. Her aim is to convert FD tissue into fully mineralized bone in a lasting and meaningful way.
As Dr. Riminucci and Dr. Yang enter the final months of their award year, FDF’s Scientific Advisory Council will be looking over the applications for the 2018 awards. Along with UPenn scientists, FDF advisors will decide the four best projects to advance our understanding of FD/MAS and improving the lives of patients. Some awarded projects might, like the last five projects, continue the trend of studying the basic science of FD/MAS with preclinical models, but at least one award has been reserved for a promising clinical science study. This will help to promote diversity in the award winning projects, ensure that the FD/MAS community is advancing as many promising research opportunities as possible, and increase the speed it takes for funded research to directly touch the lives of the FD/MAS community.
Both Dr. Riminucci and Dr. Yang agree that the Million Dollar Bike Ride grants are an invaluable resource and support for the work that they do. These grants and this incredible research would not be possible without the dedication and generosity of the FD/MAS community and their friends and families. The 2018 awardees will be announced in December.
“I knew something was not right with my child. I could sense it from when he was very small,” explained Mauricio Saravia’s mother Marisa Damele. “Then, as he grew older, he would hit his head on the wall and with his hands, we didn’t know exactly why he would do this. I’m his mother. Mothers know their children like no one else could. I knew something was not right. But the doctors would not listen to me.”
In advance of the publication of her new memoir, Marisa spoke with the FDF to share the painful and magical story of her son’s fascinating life. A painter, writer, artist and one of the most famous people with McCune-Albright syndrome, Mauricio lived a whirlwind life–sometimes glamorous, sometimes harrowing–until he passed away from complications of McCune-Albright syndrome in 2008 at the age of 39.
Marisa’s frustration with doctors mirrored that of many other FD/MAS patients and caregivers. It was hard for her to convince medical providers who were unfamiliar with the disease of the medical intricacies, and her family had to become experts out of necessity. Marisa and Mauricio’s experience was exacerbated by where they lived, the South American country of Uruguay. The practice of medicine in Uruguay when it came to FD/MAS was significantly behind that found in the United States. “Mauricio and I went on a journey, an odyssey really, to find the best care we could and make the most of this life,” said Marisa. “His determination and positivity were relentless. He truly believed he had a purpose in life and everyone who met him loved him and became a believer as well.”
When Mauricio was six years old, he traveled to the United States for surgery at Shriners Children’s Hospital in Springfield, MA. “By this time he had already lost the sight of his left eye.” recalls Marisa. “His father would bring him up for surgery, but afterwards he would need to leave and return to Uruguay while Mauricio remained in the hospital’s care for as long as 3 months at a time.” Alone and limited by his recovery, Mauricio taught himself English and began drawing to entertain himself. “Shriners was a magical place for these children, they really did their best to keep the kids happy. I think it was at Shriners that he really fell in love with the United States.”
As a child living with significant chronic pain and deformity, Mauricio dealt with terrible teasing and bullying, but he never let his limitations or looks define him. “I think many people who do not fit society’s idea of beauty tend to hide away and shrink into themselves, but Mauricio found a way to reflect and reach outward.” At nine years old, Mauricio overheard a panel of doctors say that he would would not live a long life. Years later, Mauricio said “The deadline that doctors put on my life, made me appreciate life more.” He never took time for granted and wanted to experience as much of the world as he could.
“Mauricio would go to a dance club and insist on dancing in the middle of the floor. People would say terrible things to him, but before long he’d have two or three women out there dancing with him. His enthusiasm for life was so infectious, it was impossible to be gloomy around him.”
Although he did not have the necessary immigration authorization, in his twenties Mauricio decided to move to Denver in 1998 to seek out a better life for himself. Mauricio taught himself philosophy, computer technology and design. He started writing poetry and painting, and developed his own business, freelancing as a graphic designer. “Perhaps because he looked so different, people assumed that he had to be here legally,” said Marisa. During his time in Denver, Mauricio made many close friends, launched his own magazine, Artists Interviews, where he interviewed artists and celebrities, and continued to develop his skills as an artist. Despite flourishing as an artist and creative, life in Denver was not always easy.
“Mauricio became very ill while he was in Denver and I came to the country to help him. He wasn’t able to eat or digest food, he became very dehydrated and even fell unconscious.” Marisa brought her son to a hospital where they immediately put him on IV fluids. “I was so grateful that they revived him, but the doctors told me never to set foot in the hospital again.” Because Marisa and Mauricio were undocumented, they were told that the hospital would no longer serve them, regardless of how sick he might be.
Mauricio continued to work and to pursue art, but he also struggled with his health. When his health took another turn for the worst, and it seemed that he was once again at death’s door, the hospital turned him away without care. “Thankfully, there was a pastor who was willing to help us.” They fed Mauricio small sips of water and chicken broth to slowly bring him back to life. “The Mormon church was very kind to us, they helped us and cared for Mauricio in their clinic, even though we were not Mormons ourselves.” recalls Marisa.
Mauricio eventually found more specialized care at University of California, Los Angeles. The clinic there helped him control some of the endocrine system problems that led him to be so sick.
Now living in Los Angeles, Mauricio continued to find success in his work. He attended red carpet events alongside A-list celebrities for Artist Interviews, he created movies, and continued to paint. Perhaps one of the most interesting aspects of Mauricio’s life was his interest in the glamour and beauty of Hollywood, in contrast to his deep, reflective writings on the meaninglessness of physical beauty. Much of his writing offered philosophical and poetic ruminations on the rejection of our physical forms. Even in the midst of Los Angeles’ celebrity culture, his writing, his art, and his personal connections were imbued with the depth of Mauricio’s spirit and intellect.
“He was doing so much better, I thought that he would live forever,” said Marisa, but although Mauricio’s endocrine issues were under control, his scoliosis continued to cause problems. Eventually, Mauricio was overcome by complications of untreated and severe scoliosis.
“The last few months of Mauricio’s life were very difficult. He began to have trouble eating and speaking,” said Marisa, “If there had been a willing surgeon, I know we could have saved his life, but they would ask us where the money was, where was the health insurance? And for that there was nothing we could do.” As an undocumented person, he had substantial obstacles to getting the health coverage needed to access surgical care.
Mauricio passed away in December 2008, just short of his 39th birthday. While Mauricio had severe FD/MAS, FD/MAS is not typically a fatal disease. FD/MAS-related scoliosis can be lethal, but with proper care it can usually be managed so it does not result in death.
Mauricio’s life was extraordinary for the legacy that he left behind. He is remembered through documentary film and a children’s book. His contributions to art and poetry have been recognized by multiple institutions: there is a Mauricio Saravia Art Academy in Denver, Colorado and a Mauricio Saravia Achievement Award at the Metropolitan Denver University. Every April, Metropolitan Denver University also holds a Mauricio Saravia Poetry Series.
Mauricio was extraordinary because of who he was–his boundless courage, optimism, and resourcefulness–and the effect he had on those around him. His deep spirituality, thoughtfulness, and creativity brought him many wonderful experiences and friendships. One such friend was Sean Hepburn Ferrer, film producer and son of Audrey Hepburn, who ultimately wrote the children’s book about Mauricio’s life. Sean said simply that “After meeting him, in a short time, the physical gave way to the spiritual- and there he was- in all his beauty.”
Mauricio’s life offers important perspectives: a perspective on how challenging FD/MAS can be, and how wondrous a life touched by FD/MAS can be when someone insists upon living life fully in spite of it. There’s more to learn more about Mauricio, and the incredible story of his life and journey. Keep an eye out for Marisa Damele’s upcoming memoir to learn more.
Though fibrous dysplasia/McCune-Albright syndrome (FD/MAS) is not, in and of itself, a fatal condition, the many complications associated with this chronic and rare disease can occasionally be life threatening. One example of a potentially fatal complication is scoliosis, the sideways curvature of the spine, which can affect FD patients. If left unmanaged, severe scoliosis can prove to be fatal.
A recent research paper, lead by FDF advisor Dr. Alison Boyce of the National Institutes of Health (NIH), looked at the threat of scoliosis and analyzed several methods of managing the condition. Dr. Boyce worked alongside researchers from Tufts University, Boston College, Nemours Children’s Health Center, and several other NIH researchers to study this question. They looked at 138 patients, 84 of whom had scoliosis ranging from mild (55 patients) to moderate (11 patients) to severe (18 patients).
“We found that the risk of scoliosis is highly correlated with a patient’s overall disease burden,” explained Dr. Boyce, “this means that the more bones affected by FD, the more likely that patient is to also experience some degree of scoliosis.” Other risk markers include leg length discrepancy, hip range of motion, and strength of the gluteus medius and maximus. Although the study indicates that the majority of patients likely have some degree of scoliosis, Dr. Boyce highlights there are some well-established steps FD/MAS patients can take that may prevent their scoliosis from becoming severe, including addressing leg-length discrepancies and particular hormone issues like FDF23-mediated hypophosphatemia and hyperthyroidism: “It’s important for patients to see physiatrist and also seek treatment for any endocrine disease issues which may exacerbate spine curvature,” said Dr. Boyce.
The researchers analyzed the progression of 69 patients, patients who had a series of radiographs taken over time, to learn if other treatments were useful in slowing the progression of FD/MAS. The 69 patients pursued a variety of management options, including bisphosphonates, spinal fusion (operative treatment), and no treatment, and the researchers wanted to know what outcomes were related to the different treatments. “Bisphosphonate use didn’t appear to change the progression of scoliosis for FD/MAS patients . The progression and severity of scoliosis was similar for people who took bisphosphonates and people who went untreated.” said Dr. Boyce, “There were 10 patients who were treated with spinal fusion, one of these patients did experience instrumentation failure, but 9 were able to maintain a stable spine for the 6 years we observed them. There were two fatalities in the group due to complications associated with scoliosis, and both of these patients were managed non-operatively.”
“The findings reaffirm that FD patients, especially those with polyostotic FD, should monitor bone lesion growth carefully,” Said Dr. Boyce, “There is no indication that bisphosphonates help, but spinal fusion for patients with severe scoliosis does seem to be an effective way to stop the progression of scoliosis and prevent further complications, including death.”
FDF encourages patients who are concerned about the curvature of their spine to seek the care of a knowledgeable doctor who has experience treating patients with FD/MAS. The primary risk factor identified in this study for progressive scoliosis was total disease skeletal burden (i.e. how much of your skeleton is affected by FD), underlining the importance of the recommendation in FDF’s Treatment Guidelines that FD/MAS patients receive a full body scan to understand which bones are affected by FD, and to monitor those lesions over time, rescanning every several years.
To read the full research article, or to print it out to bring to your doctor, click here.
As the FD/MAS community continues to share their stories and medical experiences through the FD/MAS Patient Registry, the Registry is now large enough to power academic research. “Participants have contributed over 100,000 data points by filling out the surveys on fdmasregistry.org,” said Deanna Portero, FDF Executive Director, “All that patient-provided data offers a wealth of information for researchers to analyze, and could lead to new and useful insights into FD/MAS.”
To help initiate new academic studies, the FDF is announcing an open call for researcher applications for use of the FD/MAS registry. “We’re in the process of contacting over 300 researchers who have already published in some capacity on FD/MAS and invited them to make use of this incredible resource,” said Portero. Researchers will receive a letter from the Foundation and an infographic that shares the power of the patient cohort that make up the Registry. “The registry really is a one of a kind resource,” says Portero, “with over 500 patients who are interested in participating in additional studies, and 300 patients providing survey data, the FD/MAS Patient Registry is one of the richest FD/MAS research resources in the world.”
”High profile FD/MAS researchers have already been in touch to let us know that they will be filing applications for data access,” continued Portero. “We hope to see the first papers based on registry data submitted for publication within the next 12 months, and we expect registry data to power research studies for years to come. The beauty of the registry is that when an FD/MAS patient completes a survey, that information can be used and reused in multiple studies. By dedicating an afternoon to participate in this one study, a patient’s story can help power many studies for years to come.”
This researcher outreach initiative was made possible by the dedication of our Spring Semester Research Intern Chidi Ezeonyebuchi and many volunteers, including Brittany Anderson, and the University of California, Berkeley’s American Bone Health club, led by undergraduate Amy Fann. Not only did Ezeonyebuchi design and create the Registry infographic, he also spent the spring engaging with Registry participants and encouraging them to keep working on their surveys. Ezeonyebuchi graduated this spring from University of Maryland and plans to apply to medical school in the near future.
Community member Brittany Anderson and volunteers from the American Bone Health club at UC Berkeley helped FDF research contact information for over 100 researchers who had published on FD/MAS but were not previously in touch with the Foundation.
The Fibrous Dysplasia Foundation is grateful for all of the volunteer work that allows the FD/MAS Patient Registry to thrive, including the assistance of the members of the FD/MAS Registry Steering Committee, the group that carefully reviews researchers requests to use the registry. This volunteer committee include researchers Alison Boyce MD, Andrea Burke DMD, MD, and Amanda Konradi PhD, and and FD/MAS community members Cindi Brandt Levin, Catherine Fairchild, and Kiran Murty.
The May 20th, 2018 Million Dollar Bike Ride marked Team Fibrous Dysplasia’s fourth consecutive year participating in University of Pennsylvania Orphan Disease Center’s charity ride for rare disease research. Team FD raised a record breaking $272,712.14 for FD/MAS research–more than the amounts raised in 2016 and 2017 combined. 100% of that sum will go directly to funding research grants for FD/MAS. This marks another exciting step in the FD/MAS community’s path towards a treatment or cure.
The ride has become a popular event for rare disease groups because it’s such a powerful way for a disease community to advance the state of research for particular diseases. “For Team FD, this year was an especially salient reminder of our power,” Said FDF Executive Director Deanna Portero, “Team FD had access to a new fundraising webpage system that made it easy for patients, families, and friends to self-enroll as fundraisers, donors and riders.” As a result, eleven campaigners stepped up alongside Team Captain Cindi Brandt Levin to advance FD/MAS research. The campaigners encouraged over 400 friends, coworkers, neighbors, and family members donate, and all those small acts of kindness and determination will lead to over $270,000 worth of research.
Among Team FD campaigners this year were several community keystones, including Cycle for Carly, Lauren Ruotolo, Rylan’s Rhythms, FD Warriors, Deanna Portero, Bike to Best FD, and Jacky’s Journey, all of whom campaigned for last year’s bike ride as well. They were joined this year by new and enthusiastic campaigners: The Healy family, A Cure for Jonathan, Hope for Harper, Wahoo Riders, and Adam’s Friends and Fans. “These patients, families, and advocates reached out and asked their communities to support our dream of better treatment options and ultimately a cure for FD/MAS patients, and our success is a testament to the generosity and commitment of all the friends and loved ones who rose to that occasion,” said Brandt Levin.
While not everyone was able to make it to Philadelphia for the actual bike ride, campaigners showed real creativity to encourage supporters to donate. The Healy family threw a “Shake Your Bones” dance party to help raise over $60,000. Top fundraisers Ziv and Orit Rosenblum of team “A Cure for Jonathan” used letter writing and phone calls to encourage donations from across the globe, including gifts from Israel, Germany, and Australia, to raise over $70,000. Another campaigner sent supporters original artwork from her 18 month old son to raise about $800 from her family and friends, and FD Warriors Inc. sold t-shirts that said “I ride for FD/MAS research” and gave the proceeds to Team FD.
“There are a million ways to fundraise to support the fight against FD/MAS,” said Executive Director Deanna Portero. “The only method that has been proven to be unsuccessful is to never try.”
Riders who did make it to the event were treated with mostly clear skies and a lovely ride along the Philadelphia’s Schuykill river. Team Wahoo Riders, led by parents Kim Indressano and Brian Romer, rode together as a family after raising over $13,000 for research. This family of four made up the largest contingent of Team FD’s 9 riders, who rode anywhere from 13 to an impressive 72 miles in support of rare disease research.
“I cannot express how thrilling it was to watch Team FD come together this year,” Said Brandt Levin. Over the last 3 years, Team FD and the FD/MAS community has raised nearly $300,000 for FD/MAS research, and this year nearly doubles that number to $560,000 directly to FD/MAS research. Together, Team FD has funded grants to explore proteins that block FD lesion growth, and the development of more accurate mouse models of FD/MAS. “These advances have been vital to the FD/MAS community’s progress towards a treatment or cure and we are thrilled to be able to offer grant opportunities to as many promising research proposals as we possibly can.” said Brandt Levin.
The type of grants that Team FD awards through the Million Dollar Bike Ride are often called “seed grants” or “pilot grants.” They are called that because these grants give researchers financial support for new ideas with great potential for high-impact, when those ideas are in the earliest stages. The researchers use the seed grant funding to generate enough preliminary data and results that they can win larger grants from institutions like the National Institutes of Health, major research foundations, or funding from a private company to pursue the new idea for an effective treatment. Biomedical research and innovation are very expensive, and some new ideas will inevitably fail, so providing ample and consistent funding for seed grants is part of FDF’s strategy to #CureFDMAS. “This fundraiser is extremely important for “seeding“ a pipeline of treatments and other innovations in research. Without these initial investments in high-risk, high-reward scientific projects, the chance of finding a “gamechanger” for FD/MAS drops dramatically,” explains Portero.
Moving forward, the FDF’s Scientific Advisory Committee will put together a request for proposals to encourage scientists to apply with their most promising research ideas to advance FD/MAS science. Grants will be awarded by UPenn with the guidance of FDF’s Scientific Advisors and announced in December of this year. FDF will continue to monitor these grants and update the community every step of the way.
The Million Dollar Bike Ride wasn’t the only charity event to support FD/MAS research this May. FD/MAS research in Holland received a boost that same weekend from the Huiseid Festival, an outdoor music and merriment festival held in Warmenhuizen, the Netherlands.
The festival includes four different stages with live music and dance performances throughout the
weekend, as well as recreational soccer and other activities available for kids. In addition to the traditional music festival, this year Huiseid also organized a pre-festival event specifically designed around the needs of those with disabilities.
Proceeds from the event go to support three different charities: the Cystic Fibrosis Foundation, Bones4Kids, and the Leiden University Medical Center through the Bontius Foundation. Through the generous support of FD/MAS patients, their friends and family, and the Huiseid Festival staff, donations to Leiden University Medical Center will go directly to research on fibrous dysplasia.
Just as the 2018 Million Dollar Bike Ride had a record breaking year, so to did the Huisweid Festival. They raised over 125,000 euros to be split between the three charities!
The FD/MAS community in the Netherlands has supported important research at Leiden University Medical Center. For example, funds the community raised were behind the analysis that established a tentative connection between FD/MAS and breast cancer, among other recently published research. The Bontius Foundation provided financial support for graduate students like Bas Majoor, who recently defended a doctoral dissertation on fibrous dysplasia.
Even though the presentation of FD/MAS can vary dramatically from patient to patient, and health systems are very different from country to country, the FD/MAS patient community as a whole has many of the same obstacles and goals the world over. It is thrilling to see the Dutch community come together at this fun event in support of research on FD/MAS.
For the last four years the FD/MAS community has been REALLY excited about a particular bike ride in Philadelphia. It’s not that we’re super cyclists, it’s just that this event happens to be the biggest driver of private FD/MAS research funding in the world. Through the Million Dollar Bike Ride, Team Captain Cindi Brandt Levin and the community of amazing fundraisers who setup fundraising pages have led the FD/MAS community to raise $300,000 for FD/MAS research. Thanks to these grants, there have already been some really promising breakthroughs, but our continued participation in the Million Dollar Bike Ride isn’t guaranteed.
The Million Dollar Bike Ride is organized by UPenn School of Medicine’s Orphan Disease Center and they invite many rare disease communities to participate. UPenn matches the money each disease team raises up to $50,000. That’s like a free $50,000 towards a treatment or a cure for any community that’s organized enough to rally and raise the funds. The event has become really popular, so popular that they have now decided to limit the number of diseases that compete. So how will they choose which rare disease communities get to join the ride and access up to $50,000 in free research dollars?
Only the top 20 teams from this year’s race will get a spot in the 2019 Million Dollar Bike Ride. Team FD will be ranked against other racing teams by the number of dollars we raise, and the number of cyclists who ride in the race.
Last year, Team FD had more people involved in our efforts than ever before. Seven families set up crowdfunding pages, 12 cyclists actually rode on the day of the ride, and over 200 people donated. All these people helped Team FD raise $107,228 for FD/MAS research! To keep funding grants through the Million Dollar Bike Ride in 2019 and beyond, we’ll need even more community members to get involved as riders, fundraisers and donors.
Deanna Portero, executive director of the Fibrous Dysplasia Foundation was one of the cyclists in the 2017 race. “Everyone at the race had a positive experience. I’m not an athlete at all, so I chose the short and flat 13-mile route. It was inspiring to ride through Philadelphia on a nice spring day while fueling FD/MAS research.” This year, Deanna will be asking a few friends who live in Philadelphia to join her in the race. “After the race there is usually a great buffet and selection of local beers for riders sponsored by UPenn, which is a great time too.”
FD/MAS parent and FDF Board Member Jocelyn Pederson also joined Team FD for the first time last year. “We were surprised at how easy it was,” said Jocelyn,“ Our friends and neighbors just want to help and the Million Dollar Bike Ride gave them the tools to do that. It was incredible, the outpouring of support we felt.” Jocelyn couldn’t come to Philadelphia to bike, but by creating a fundraising page she took an even more crucial step in supporting Team FD, and FD/MAS research.
“Many people find success fundraising for the Million Dollar Bike Ride, because every single cent of every single donation goes directly to research grants. Plus, University of Pennsylvania truly does match the donations from FD/MAS patients, family and friends. Donors can make double the difference that they normally world” explained Deanna.
At this very moment, Team FD research grants are powering ground breaking research in Rome and Boston. Last year, Dr. Mara Riminucci shared with us that her mouse model studies showed a complete stop in FD bone growth, and now she’s working on perfecting the medication dosage for the mouse so she can eventually move to human trials. This progress could not happen without the brave people who joined Team FD!
“Every time a patient or family member makes a donation, even a small one, they are becoming a part of this push for a cure,” explains Cindi Brandt Levin, “We need as many people joining this cause as possible.” Starting a campaign is easy; you can set your fundraising goal and choose whether or not you want to go to the actual Bike Ride. Have questions or need help getting started? Just ask us!