As long as we are here, no patient need ever face this disease alone. The FSHD Society is deeply committed to working with ingenuity and integrity to accelerate research leading to treatments and a cure.
It is the generous support - gifts of time, talent, and treasure - from patients, families, friends, major donors, and sponsors, that make this imperative work possible.
Our Core Values
Research | Community | Urgency
The FSHD Society is the world’s largest research-focused patient organization for facioscapulohumeral muscular dystrophy (FSHD), one of the most prevalent forms of muscular dystrophy. We have catalyzed major advancements and are accelerating the development of treatments and a cure to end the pain, disability, and suffering endured by one million people worldwide who live with FSHD. The FSHD Society has transformed the landscape for FSHD research, and is committed to making sure that no one faces this disease alone.
Our Focus Areas
Accelerating research toward treatments
- Therapeutic Accelerator. We bring all of the stakeholders together to focus on removing obstacles to the success of clinical trials and regulatory approval of FSHD therapies.
- Clinical Trial Readiness Initiatives. We invest in infrastructure to position the field for clinical trials. Priorities include the Clinical Trial Research Network and programs to develop “trial ready” patient communities.
- Research Funding. We award grants to the best basic research to drive understanding of the disease, identify therapeutic targets, and develop the tools needed to translate discoveries into treatments.
- International Research Congress (IRC). We gather the world leaders in FSHD research every year to promote rapid communication of research advances, encourage collaborations, and set research priorities for the future.
Activating and empowering our stakeholders
- Strengthening communities. We bring patients and families together to support one another and share knowledge. We train and empower volunteer leaders to become effective advocates and fundraisers in their communities.
- Knowledge is power. We publish authoritative news and brochures, and organize meetings to educate patients and families about diagnosis, managing symptoms, and clinical research. These include our national chapter program, FSHD 360 conferences, and FSHD University, a patient education program.
- No one need face this disease alone. We are available 24/7 for patients and families via phone, email, and social media. We facilitate a thriving chapter program across North America and an online community we call the Gathering Place, volunteer-run groups with thousands of members..
- Advocating for action. Our co-founder Daniel Perez serves on the presidential Muscular Dystrophy Coordinating Committee which meets annually to assess progress on all of the muscular dystrophies. We work closely with NIH and FDA officials, and advocacy organizations worldwide to push forward policies and legislation to help our community.
The FSH Society has funded over $12 million in research initiatives that have propelled us into the era of treatment discovery and development. Our work has contributed to identifying the genetic mechanism of FSHD, development of animal models, validation of clinical trial endpoints, and early-stage drug discovery research. Other signature achievements include co-authorship of the 2001 MD CARE Act that mandated federal attention to all of the muscular dystrophies and led to the NIH-funded Wellstone Cooperative Centers for Muscular Dystrophy Research; the 2015 publication by the National Academy of Neurology of the first evidence-based FSHD care guideline; helping to launch the nationwide FSHD Clinical Trial Research Network in 2016; and the landmark Voice of the Patient Report, which is considered by the FDA when it reviews new FSHD therapeutics. For more details, please see our annual Donor Impact Reports.