“Never doubt that a small group of thoughtful, committed citizens can change the world: indeed, it's the only thing that ever has.”
- Margaret Mead, anthropologist
This is a historic time in FSHD research. Therapies to slow down or stop the progression of symptoms are being tested for the first time in patients. More drug candidate are on the way. Based on a realistic—and aggressive—assessment of the state of research, we have pledged to deliver a treatment to our families by the year 2025—if not sooner.
To receive e-alerts about FSHD clinical studies and trials, please join the Research Contact Registry.
Status update on disease-modifying therapies
Around a dozen biopharmaceutical companies and academic laboratories are developing treatments for FSHD. The lead prospects range from drugs that tamp down the expression of the DUX4 gene—thought to be the key trigger for muscle degeneration in FSHD—to gene therapies that target DUX4 messenger RNA. Fulcrum Therapeutics will be conducting a Phase 3 trial in 2022. Avidity Biosciences, Arrowhead Pharmaceuticals, and Dyne Therapeutics have announced plans to launch clinical trials in 2022-2023.
Questions? If you’d like to learn about research opportunities, locally and nationally, or would like specific ideas about how to get involved, Contact Us.
We provide guidance on how to talk about your diagnosis with your healthcare providers, your family, friends, coworkers, and the world at large.
Keep this page handy. It’s your “FSHD 101” with resources at your fingertips on symptoms, genetic testing, self-care and health care.
There’s comfort and power in numbers. Individuals and families in your local area can be an invaluable resource. We’ll connect you.