Fibrous Dysplasia Foundation, Inc.
Research. Advocate. Collaborate.
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Programs and results
What we aim to solve
Our programs
What are the organization's current programs, how do they measure success, and who do the programs serve?
Global Rare Disease Registry of Patients with Fibrous Dysplasia/McCune Albright Syndrome
The Global Rare Disease Registry of Patients is a research project that allows patients and families to share their experiences with Fibrous Dysplasia/McCune-Albright syndrome (FD/MAS) by completing a series of surveys. The FD/MAS Patient Registry is a centralized source of information on FD/MAS. We encourage researchers to utilize the registry.
FD/MAS Research
The Alliance raises money for seed grants through The Million Dollar Bike Ride hosted by Penn Medicines Orphan Disease Center. Seed grants are awarded to researchers or research teams to develop and test new ideas or concepts to advance scientific knowledge about fibrous dysplasia and McCune-Albright Syndrome. Team FD/MAS was once again the top fundraising team in 2023, and together, we raised $161,284.
The Alliance has also successfully advocated for the inclusion of FD/MAS research in the Peer Reviewed Medical Research Program (PRMRP) of the Department of Defense for the past four years.
In 2023, FD/MAS Researchers received more than $6.7 million in federal funding
A grant for $3,346,788 was awarded by the Department of Defense to Drs. Yingzi Yang, and Marc Wein
An R01 Grant for $3,360,000 was awarded by (NIH) to Drs. Edward Hsiao; Fernando Fierro, Kelly Wentworth, and Richard Nissenson, PhD
Where we work
External reviews
Videos
Our results
How does this organization measure their results? It's a hard question but an important one.
Evaluation documents
Download evaluation reportsNumber of research studies funded
This metric is no longer tracked.Totals By Year
Type of Metric
Output - describing our activities and reach
Direction of Success
Increasing
Context Notes
Rare disease research is a marathon, not a sprint. FD/MAS Alliance works to raise money with the University of Pennsylvania Orphan Disease Center and other partners to ensure that grants are awarded.
Number of participants engaged in programs
This metric is no longer tracked.Totals By Year
Related Program
Global Rare Disease Registry of Patients with Fibrous Dysplasia/McCune Albright Syndrome
Type of Metric
Output - describing our activities and reach
Direction of Success
Increasing
Context Notes
The FD/MAS Patient Registry continues to grow in participant numbers, and data contributed, solidifying it as a valuable resource for FD/MAS research.
Our Sustainable Development Goals
Learn more about Sustainable Development Goals.
Goals & Strategy
Learn about the organization's key goals, strategies, capabilities, and progress.
Charting impact
Four powerful questions that require reflection about what really matters - results.
What is the organization aiming to accomplish?
The name of this condition, Fibrous Dysplasia/McCune-Albright syndrome (FD/MAS), can sometimes be confusing. Some people only have affected bones, which might be called FD. When bones and other tissues are affected, it is called MAS. Since the same gene mutation causes both FD and MAS and since many people with FD also have other tissues involved, the FD/MAS Alliance and doctors have decided to call it FD/MAS. It is a rare condition caused by a mutation in the gene GNAS. The mutated gene produces a protein, Gs alpha, that is dysfunctional. Gs alpha is an on/off switch in cells, and the mutated protein is stuck in the on position. Depending on the cell or tissue affected, the on switch has a different effect.
The tissue most commonly involved is the bone (FD). Here, the mutated protein makes it so bone cells aren't able to mature into normal bone. When endocrine organs, such as the thyroid or ovary, are affected, the effect of the on-switch is to make excess hormones. Certain skin cells produce pigment, leading to darkened skin, sometimes called caf-au-lait macules. Since the Gs alpha protein is produced in almost every cell in the body, many organs can be involved.
As a community-led patient advocacy organization, our ultimate goal is that
1. Individuals affected by FD/MAS have widespread and comprehensive access to effective, evidence-based treatments for endocrine, craniofacial, neurological, orthopedic, and other complications associated with the disease.
2. Cutting-edge treatments and, ultimately a cure for FD|MAS are discovered and are widely available to affected individuals.
The FD/MAS Alliance's mission is to develop evidence-based treatments and foster the well-being of individuals with FD/MAS by advancing cutting-edge research, channeling the voices of individuals with FD/MAS, and sharing information with the medical community.
What are the organization's key strategies for making this happen?
1. Maintain a comprehensive, up-to-date repository of information for patients, caregivers,
and medical practitioners, including recent research publications, diagnosis and treatment guidelines, and vetted clinicians with experience treating the disease.
2. Create opportunities and mechanisms for information-sharing and mutual support,
including the development of written resources for patients and medical professionals, responses to individual inquiries by FD|MAS staff, and venues (such as conferences, webinars, and social media) for patients, caregivers, clinicians, and researchers to interact virtually and in-person.
3. Coordinate and advance the funding of clinical research on promising treatment practices, techniques, and therapies. (e.g., Congressionally-directed funding programs)
4. Facilitate the funding of basic science research and interdisciplinary research on the
molecular and cellular mechanisms of the disease and their application to treatment.
5. Expand the use of the patient registry by researchers seeking to improve diagnosis and care
and to test promising treatments, techniques, and therapies.
What are the organization's capabilities for doing this?
FD/MAS Alliance is in a critical growth phase, building more sustainable and professional infrastructure to improve our ability to deliver our mission effectively now and in the future. We recognize that capacity building will be conducted over time, and we have identified which items we need to work on to be most successful at the current stage of our lifecycle as an organization, namely Strategic planning and leadership development, program measurement and evaluation, and building financial capacity.
Focusing on these areas will allow us to strengthen our board and staff leadership, further develop programs, adopt best practices, and ignite our fundraising efforts. Doing so will pay off in other dividends.
Initially, we anticipate hiring consultant(s) and a consulting firm to assess the organization, facilitate a strategic planning process, and provide board and staff leadership training. We also intend to synthesize existing knowledge, measure and evaluate the effectiveness of our programs, and activate our new patient advisory panel to attract new participants to programs and the patient registry. Finally, we are excited to embark on efforts to increase our financial capacity by hiring outside assistance for grant writing and other practical and sustainable fundraising efforts to hire in-house fundraising staff.
What have they accomplished so far and what's next?
The FD/MAS Alliance has enhanced its patient advocacy and service delivery by implementing outstanding programming, thanks to the support of generous funders. In Sept 2023, FD/MAS Alliance hoted FD/MAS patients, caregivers, and investigators joined forces at a two-day conference near Washington, DC. The event in Silver Spring, MD, featured patient evaluations, informative lectures, and chances for everyone to connect.
In 9 years, Team FD/MAS has raised over a million dollars for FD/MAS research through the UPenn Orphan Disease Centers Million Dollar Bike Ride (MDBR) thanks to Team Captain Cindi Brandt Levin, the Levin family, the FD/MAS Alliance, & the FD/MAS community. These research grants are focused on developing a deeper understanding of the disease and finding better treatments! Team FD/MAS was once again the top fundraising team in 2023, and together, we raised $161,284
The Alliance has also successfully advocated for the inclusion of FD/MAS research in the Peer Reviewed Medical Research Program (PRMRP) of the Department of Defense (DOD) for the past four years. This important work has led to a successful FD/MAS grant from the DOD for the first time.
In 2023, FD/MAS Researchers received more than $6.7 million in federal funding.
- A grant for $3,346,788 was awarded by the Department of Defense (DOD) to Yingzi Yang, MD, and Marc Wein, MD, for a four-year study of Fibrous Dysplasia (FD).
- An R01 Grant for $3,360,000 was awarded by the National Institutes of Health (NIH) to Edward Hsiao, MD; Fernando Fierro, MD; and Kelly Wentworth, MD, and Richard Nissenson, PhD for a 5-year study of FD treatment.
We continue to expand our patient registry with an IRB-compliant platform; maintain and refresh a comprehensive, up-to-date repository of information; expand access to recent research publications, diagnosis, and treatment guidelines; and provide a searchable list of clinicians with experience treating the disease.
The current registry, maintained by the National Organization of Rare Disorders (NORD), will be updated in the coming year. The registry creates a cohort of interested patients ready to participate in clinical trials and other research. It also creates a body of data that can be mined for evidence-based treatment recommendations. Our goal is both to increase diversity and representation among participants and to increase patient participation levels overall.
We provide information that ensures patients are well-equipped for their doctor appointments using the Clinical Pathways (CPW) program, tools that are used to guide evidence-based healthcare. We anticipate that the CPW for FD/MAS will be updated to become more precise and accurate in the next year. FD/MAS plans to update content for patients and clinicians to ensure that it is meaningful and relevant to the guidelines and that patients are aware of this content and can find it easily.
How we listen
Seeking feedback from people served makes programs more responsive and effective. Here’s how this organization is listening.
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How is your organization using feedback from the people you serve?
To make fundamental changes to our programs and/or operations, To inform the development of new programs/projects, To identify where we are less inclusive or equitable across demographic groups, To strengthen relationships with the people we serve, To understand people's needs and how we can help them achieve their goals
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Which of the following feedback practices does your organization routinely carry out?
We collect feedback from the people we serve at least annually, We take steps to get feedback from marginalized or under-represented people, We aim to collect feedback from as many people we serve as possible, We take steps to ensure people feel comfortable being honest with us, We look for patterns in feedback based on demographics (e.g., race, age, gender, etc.), We look for patterns in feedback based on people’s interactions with us (e.g., site, frequency of service, etc.), We engage the people who provide feedback in looking for ways we can improve in response, We act on the feedback we receive, We tell the people who gave us feedback how we acted on their feedback, We ask the people who gave us feedback how well they think we responded
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What challenges does the organization face when collecting feedback?
It is difficult to get the people we serve to respond to requests for feedback, It is difficult to find the ongoing funding to support feedback collection, Staff find it hard to prioritize feedback collection and review due to lack of time
Financials
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Operations
The people, governance practices, and partners that make the organization tick.
Connect with nonprofit leaders
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- Analyze a variety of pre-calculated financial metrics
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Connect with nonprofit leaders
SubscribeBuild relationships with key people who manage and lead nonprofit organizations with GuideStar Pro. Try a low commitment monthly plan today.
- Analyze a variety of pre-calculated financial metrics
- Access beautifully interactive analysis and comparison tools
- Compare nonprofit financials to similar organizations
Want to see how you can enhance your nonprofit research and unlock more insights? Learn More about GuideStar Pro.
Fibrous Dysplasia Foundation, Inc.
Board of directorsas of 01/23/2024
Ms. Anne Corvelle
Malissa Kenney
Gary Pollastro
Anne Corvelle
Cindi Brandt Levin
Justin Healy
Lauren Ruotolo
Carmel Shemmesh-Rafalowsky
Eric Liao, MD
Justin Cohen, MD
Board leadership practices
GuideStar worked with BoardSource, the national leader in nonprofit board leadership and governance, to create this section.
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Board orientation and education
Does the board conduct a formal orientation for new board members and require all board members to sign a written agreement regarding their roles, responsibilities, and expectations? Yes -
CEO oversight
Has the board conducted a formal, written assessment of the chief executive within the past year ? Yes -
Ethics and transparency
Have the board and senior staff reviewed the conflict-of-interest policy and completed and signed disclosure statements in the past year? Yes -
Board composition
Does the board ensure an inclusive board member recruitment process that results in diversity of thought and leadership? Yes -
Board performance
Has the board conducted a formal, written self-assessment of its performance within the past three years? Not applicable
Organizational demographics
Who works and leads organizations that serve our diverse communities? Candid partnered with CHANGE Philanthropy on this demographic section.
Leadership
The organization's leader identifies as:
Race & ethnicity
No data
Gender identity
Transgender Identity
Sexual orientation
No data