FD/MAS Research Priorities Workshop: Recap
Community and Science Came Together at the FD/MAS Research Priorities Workshop
Patients, caregivers, clinicians, academic researchers, and pharmaceutical scientists gathered in Philadelphia for a landmark day, dedicated entirely to charting the future of fibrous dysplasia and McCune-Albright syndrome (FD/MAS) research.
The FD/MAS Research Priorities Workshop, organized by the FD/MAS Alliance and chaired by Scientific Advisory Council Chair Dr. Michael Collins, brought together a diverse and deeply invested community with a shared mission: to identify what matters most to the people living with FD/MAS and to align the scientific community around those priorities.
105 Community Survey RespondentsA Room of Key Stakeholders
One of the most meaningful aspects of the workshop was who was in the room. The 63 in-person attendees included 15 providers, 12 pharmaceutical researchers, 19 academic researchers, and 26 patients and caregivers — many of whom wore more than one hat. The gathering was shaped by voices from around the world, with clinical and basic science speakers joining from the Children’s Hospital of Philadelphia, the National Institute of Dental and Craniofacial Research, the University of Claude Bernard in France, Sapienza University in Rome, Boston Children’s Hospital, and Sichuan University in China.
Critically, community members were not just observers — they were speakers. FD/MAS Alliance Vice President Nicola Deeley Cannings, President Anne Corvelle, adults living with FD/MAS Kara LeClaire and Richard Pace, and Patient Advisory Council members Camryn Berry and Tyler Healy all took the floor, grounding every scientific discussion in lived experience.
Six Priorities That Reflect What the FD/MAS Community Needs Most
Through structured discussion and guided sessions, the group coalesced around six research priority areas that span the breadth of what the FD/MAS community faces day to day:
- Pain Management: Effective treatments and better education for care teams on managing chronic pain in FD/MAS patients.
- Treatment Options: Novel therapies, repurposed medications, and non-pharmaceutical approaches to managing the disease.
- Craniofacial Care: Dedicated treatment options for the craniofacial manifestations of FD/MAS, which affect many patients profoundly and seem to be different than FD lesions in other areas of the body.
- Scientific Understanding: Deepening knowledge of FD/MAS biology and its effects across multiple body systems beyond bone.
- Reproductive Health: Research into fertility, menopause, and the impact of existing treatments on these life stages.
- Access to Knowledgeable Care: Ensuring patients everywhere can access clinicians who understand and are equipped to treat FD/MAS.
There is a renewed interest in focused, collaborative discussions that can unlock state and federal funding, encourage collaborative research efforts, and advance the outcomes most important to the FD/MAS community.
A Format That Worked
Post-event surveys reflected strong enthusiasm for how the day was structured. Attendees praised the balance between community voices, scientific presentations, and open discussion. The sense of belonging fostered throughout the day was highlighted repeatedly — a reminder that for a rare disease community, simply being in a room with others who truly understand is meaningful in itself.
