Resources and Funding Opportunities
This page was developed as a resource to researchers who are seeking to enter the field of FD/MAS research. We hope researchers, industry and other important stakeholders will use this overview of the FD/MAS research landscape to form new collaborations and accelerate the rate of progress in the fight to #CureFDMAS.
This page was compiled by layperson staff and volunteers for the FD/MAS Alliance. Efforts were taken to be inclusive and accurate. If you have feedback on this page, including if it was useful to you, or if there are recommended changes or additions, please contact firstname.lastname@example.org.
Centers with large cohorts of FD/MAS patients and/or biospecimens
The FD/MAS Patient Registry is a research project that allows patients and families to share their experiences with FD/MAS by completing a series of surveys. The registry launched in November 2016 and as of October 2020, more than 900 patients have joined the registry, including more than 750 who have consented to be part of the web-based study. Researchers can request de-identified datasets, request recruiting assistance, or petition for the inclusion of new measures in the registry. Researchers can learn about the registry here. For questions or feedback, contact FDF Board Member and Registry Primary Investigator Kiran Yarlagadda, email@example.com, and Executive Director Adrienne McBride, AMcBride@fibrousdysplasia.org.
The Skeletal Disorders and Mineral Homeostasis Section of the National Institute of Dental and Craniofacial Research (NIDCR) at the National Institutes of Health (NIH) runs a natural history for patients with polyostotic fibrous dysplasia or McCune-Albright syndrome. Resources include medical records, clinical data, and a repository of biospecimens from FD/MAS Patients. The purpose of the study is to define the natural history of the disease by following FD/MAS subjects over time and by using in vitro experimentation with samples/tissue from subjects with the disease. The study started in 1998 and has an estimated patient cohort of 500 patients, as of May 2018. Best points of contact are Lori Guthrie, firstname.lastname@example.org, and Alison Boyce, email@example.com. Learn more here.
The Centre for Bone Quality at Leiden University Medical Center (LUMC) in the Netherlands is currently undertaking several scientific studies into FD. Some of the research topics include: the genetic features of FD and its course, the effects of risedronate in the treatment of FD, the effect of different operations and the use of various implants and donor bone on complaints and the course of the disease, the placing of special implants that are tailored for FD patients, factors that can influence the disease, and predictors of disease activity in FD. Learn more about that center here. Best point of contact is Natasha Appelman-Dijkstra, N.M.Appelman-Dijkstra@lumc.nl.
The Institut national de la santé et de la recherché medical (INSERM) at Universite de Lyon in France is studying the relationship between bone and certain chronic diseases. INSERM has medical records and clinical data on approximately 400 patients, with approximately 250 still on follow-up. For more information please visit: LYOS. Best point of contact is Roland Charpurlat, firstname.lastname@example.org.
Rudy Study is a study of rare diseases of the bones, joints, and blood vessels led by researchers at the University of Oxford in the United Kingdom. One of the diseases included in that study is FD/MAS. The study links medical records, clinical data, donated biospecimens, and patient-reported outcomes information for enrolled participants. An estimated 167 patients with FD or MAS are currently participating in the RUDY Study. Learn more here. Best point of contact is Kassim Javaid, email@example.com.
Dedicated Funding for FD/MAS Research:
In partnership with the Orphan Disease Center at the University of Pennsylvania, the Fibrous Dysplasia Foundation offers seed grants annually. Letters of Intent (LOIs) are typically due in late summer. Email firstname.lastname@example.org to request alerts when funding becomes available. To check for current information, visit http://www.med.upenn.edu/orphandisease/rare-disease-overview.html.
Researchers investigating fibrous dysplasia have received extramural funding from a variety of NIH institutes and programs. Depending on an investigator’s research interests, the Fibrous Dysplasia Foundation encourages researchers to review funding opportunities from the National Institute of Dental and Craniofacial Research, the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the National Institute of Diabetes and Digestive and Kidney Diseases, the National Center for Advancing Translational Sciences, the National Institute on Deafness and Other Communication Disorders, the National Eye Institute, or other relevant NIH Institutes/Centers. In the past, researchers studying FD/MAS have received R01, R15, R21, R56, F32, and other forms of extramural funding from NIH Institutes/Centers.
Rare Disease Research Funding:
There are a number of grant programs that specifically seek to fund research on rare diseases.
- The Patient-Centered Outcomes Research Institute (PCORI) has a special interest in funding research on rare disease comparative effectiveness research, and programmatic activities to improve the capacity of rare disease communities to conduct more powerful, patient-centered comparative effectiveness research. PCORI funding was instrumental in previous FD/MAS projects, such as steering patients and families to the online portal of the FD/MAS Patient Registry.
- The Rare Disease Foundation, in Vancouver, British Columbia, has collaborated with the B.C. Children’s Hospital Foundation to provide microgrants of up to $5,000 CAD to researchers and clinicians to expedite the exploration of new ideas for understanding rare diseases and improve patient care. Find out more about the Microgrant Program Application here (https://rarediseasefoundation.org/pages/microgrant-program-application).
- Findacure in Cambridge, United Kingdom, and CuresWithinReach in Chicago, Illinois, are nonprofits that fund drug repurposing/repositioning research, often in coordination with each other.
- The U.S. FDA Office of Orphan Products Development funds development of pediatric devices. The nonprofit consortia provides a platform of experienced regulatory, business planning, and device development services (such as but not limited to intellectual property advising; prototyping; engineering; laboratory and animal testing; grant-writing; and clinical trial design) to help foster and guide the advancement of medical devices for pediatric patients. In addition, the consortia can provide a maximum of $50,000 per year for a Pediatric Device Consortia-supported device project. For more information on the network of consortia, click here.
- Fondazione Telethon in Italy (https://www.telethon.it/en) has a special interest in funding research on rare genetic diseases including FD/MAS. The foundation says it has already funded more than 2,700 projects and 1,600 researchers. Researchers working in public or private non-profit Italian research institutes are eligible to apply.