Light the Fire Within

Industry Collaborative for Drug Development in FSHD - Our inaugural meeting was held in 2019; a follow-up meeting will take place in June. These efforts have led us to now be working with over 15 biotech & pharmaceuticals, actively working on FSHD solutions. We continue to facilitate clinical trial readiness and patient engagement, which has expedited advisory and trial recruitment. With 3–4 trials set to launch within the year, we are increasing our efforts to ensure effective therapies can get through the process.

Voice of the Patient Forum with the FDA - We successfully engaged more than 600 patients and family members to give testimony on life with FSHD. The follow-up Voice of the Patient Report has already served as a catalyst to move therapies forward and will continue to have impact as more pharmaceuticals begin trials in the near future.

FSHD Clinical Trial Research Network (CTRN) - Currently 18 sites in the U.S. and Europe, with two more in the process of being added in Canada, work to remove barriers to getting an effective therapy to patients. Bold steps taken early on launched, then expanded the CTRN. This has been an invaluable catalyst of clinical research into trial-readiness and developing and evaluating tools to measure disease progression with an effective treatment for FSHD.

Therapeutic Accelerator Initiative - In addition to funding the best scientifically meritorious research from a growing group of investigators (grants to 40+ researchers worldwide), we have launched projects of our own.

• • TestFSHD will provide less expensive, more readily accessible genetic testing;
  • We are actively seeking to validate blood biomarkers;
  • Later this year we will launch an Health Economic & Outcomes Research (HEOR) project to quantify the cost of living with FSHD;
  • And we catalyzed the MOVE study (through the CTRN) while working with industry and academia to evaluate MRI as a potential biomarker.

Together, we have accelerated clinical and therapeutic development while investing almost $8 million over the last 3 years in targeted projects to expedite treatments to our families.

Hundreds of volunteers continue to give their time, talent, and treasure to ensure no one need face this disease alone. With 33+ chapters across the U.S., and now Canada, being led by committed, passionate volunteers, we have had immeasurable impact. Our chapter program is igniting a nation of individuals who raised millions for a cure, conduct educational and community-building events, engage local networks, and meet the core needs of our families and caregivers.

Even more is being accomplished because of you - members of a collective who have used the fire within to ignite others (all during a pandemic that largely shut down the global community.) Arnold Glascow once said, “Success isn’t a result of spontaneous combustion. You must set yourself on fire.”

In the next three years, we can get safe and effective treatments to our families. Through the intentional, deliberate act of “setting ourselves on fire” to ignite a global movement, we can build a stronger, clinical-trial-ready community and infrastructure.

We can and will continue to accelerate therapy development.

We can and will put an end to FSHD!