This is how we win

Every move we make drives us toward our goal

By June Kinoshita, FSHD Society

There is so much going on at the FSHD Society that for someone observing from the outside, it may seem overwhelming and unfocused. In truth, everything we do is intentional and part of a strategy to improve lives for people in our community and speed up the development of treatments.

The FSHD Society’s approach to combating facioscapulohumeral muscular dystrophy (FSHD) can feel like a chess game, with many pieces deployed across the board. Each is a critical player working in concert with the others to achieve the ultimate prize: effective treatments and healthier lives for everyone with FSHD.

Our strategy is two-pronged.

On the one hand, under the banner of the Therapeutic Accelerator, we orchestrate efforts to speed up clinical trials, clear the path to regulatory approval, and steer future treatments past obstacles such as lack of insurance coverage and access to care.

On the other hand, our education and engagement programs are priming our community to mobilize rapidly for natural history studies, clinical trials and, recently, what we call treatment readiness.

Here’s our game plan to ensure that our efforts are efficient and targeted to the needs of our families.

The Society’s support for the Clinical Trial Research Network and Global Innovation Hub provides the critical infrastructure for clinical research and treatment trials.

This infrastructure makes it possible to conduct efficient research on molecular biomarkers, imaging biomarkers, functional measures, and new treatments.

None of this research can move forward without broad engagement by the community. This is where our chapters, Walk & Rolls, conferences, and Zoom meetings shine.

The community now has powerful new tools with BetterLifeFSHD and the Care Connector, which link individuals to valuable resources and enable them to contribute their data to the collective effort.

These efforts must spread internationally, because FSHD doesn’t respect national boundaries—and because solutions require all of us working together. Enter Project Mercury to give patients a powerful voice with regulatory bodies like the FDA and European Medicines Agency, as well as with insurance payors and healthcare systems.

This is also why we are working on international care guidelines to provide updated medical care for patients everywhere.

Through these coordinated moves, the Society aligns all stakeholders, working together to put FSHD into checkmate.