Overcoming Obstacles - Donor Impact Report
Regardless of what is happening in the world, facioscapulohumeral muscular dystrophy (FSHD) does not stop its devastating progression, and neither can we. Your involvement, your support, and your commitment to the FSHD Society, and our collective mission, ensures that no one need ever face this disease alone.
In 2020, working together, we launched an aggressive initiative to accelerate therapeutic development in FSHD. Because of your commitment and investment, we hosted the first Industry Collaborative Workshop for Therapy Development in FSHD with pharmaceutical companies, researchers, and regulatory and governmental agencies to identify the major obstacles to clinical trial development and regulatory approval of effective therapies for FSHD.
While we expand our reach and strengthen the FSHD Community in our efforts to ensure no one need ever face their FSHD journey alone, we remain committed to bringing treatments to patients and shortening the road to a cure.
Advancing FSHD Research
Clinical Trial Research Network The nationwide FSHD Clinical Trial Research Network, launched in 2016 with funding by the FSHD Society, is a cornerstone of our strategy to accelerate the development of treatments. Comprised of leading FSHD research centers, the CTRN functions as a “lab without walls” with uniform standards and training for assessing patients and sharing data.
International Research Congress The International Research Congress (IRC), widely seen as the premier global platform for discussion and dissemination of state-of-the-art research on FSHD, is held annually to help promote and maintain worldwide collaborations and broaden accessibility to the latest research discoveries. This year’s event was held virtually.
Biomarker Project Launch On June 3rd, 2020, the FSHD Society hosted a virtual workshop to sort through and prioritize DUX4-regulated genes potentially detectable in the circulation. The Society funded testing of available commercial antibodies to a select marker, which helped identify usable tools for generating customizable assays. In addition, existing commercial assays were found for several DUX4-regulated genes. The Society is funding several studies to determine the utility of these for FSHD studies. To date, one candidate showed promising results and additional validation experiments are ongoing.
The purpose of the circulating biomarker initiative is to develop non-invasive blood-based assays to replace invasive muscle biopsies currently used to guide drug dosing and effectiveness in clinical studies.
Voice of the Patient Forum On June 29, 2020, the FSHD Society convened the Voice of the Patient Forum, our community’s externally led patient-focused drug development (EL-PFDD) meeting to educate the FDA and other stakeholders on how FSHD has impacted your life or the life of your loved ones.
The report resulting from this meeting has been submitted to the FDA and will be used in agency reviews of future FSHD therapies.
Maximizing Your Personal Strengths
Sequester Camp At the beginning of the pandemic there was so much uncertainty, and we wanted our community to know that they were very much in our thoughts. Many were feeling isolated, and it was even harder to get out, or for others to visit. The fragile webs of support that our families had been built were being shredded. So our Sequester Camp began—every Thursday, from March through May, a new webinar was presented.
FSHD University FSHD University is your center for advanced education about the art and science of living with FSH muscular dystrophy. We focus on physical health, wellness, and research, drawing on the many resourceful, creative people in our community who have earned their MDs and PhDs in living with FSHD.
FSHD Family Days An up close and personal opportunity to find out about exciting advances in research on FSHD treatments, along with essential information about staying in good shape. Learn what you can do to make a difference for yourself and for your community.
Chapter & Other Meetings/Programs In 2020, the FSHD Society maintained our commitment to bringing our community together—albeit virtually in most cases—by holding 62 chapter meetings, 29 educational programs and 27 fundraising events.
Deepening the FSHD Community
Connect Conference The 2020 FSHD Connect Classroom was held on Saturday, June 27, 2020, as a one-day online conference, to take the place of our biennial FSHD Connect conference. The conference was a great success, with more than 400 registrants who enjoyed a series of stellar presentations by medical and research superstars, with the latest news on medical care, drug development, and our nationwide initiatives.
Volunteer Led Chapters When we come together, each bringing our unique strengths, we inspire each other to go farther and reach higher than we can when we go it alone. Led by volunteers and supported by FSHD Society staff, the Chapter Program is our greatest opportunity to fund more research, connect more patients, and advance more progress.
Virtual Walk & Roll to Cure FSHD We held our first ever virtual Walk n Roll, and it was a huge success with 24 locations, 20 sponsors, 147 teams, 822 participants, 3,600 participants/supporters, and over $554,000 raised.
Newly Identified In 2020 3,848 people joined the FSHD community by signing up with their email and receiving our communications.
Newly Engaged Last year 2,113 people became new donors or attended their first program or fundraising event.