FSHD Therapeutics

A treatment accelerator initiative of the FSH Society

You can help us deliver treatments and a cure to our families by 2025.

There comes a time in the journey of every research-focused patient advocacy organization when decades of effort finally come to fruition. All the investment of money, labor, and material plowed into research cracks open the disease mechanism, and now the race begins to develop a treatment. The FSH muscular dystrophy community has arrived at this tipping point. To rise to the challenges of this new era, the FSH Society—for 27 years the global leader in advocating for research on FSHD—is launching a new initiative―FSHD Therapeutics, LLC.

Our challenges

  • Reduce the barriers to drug development in FSHD
  • Better understand disease progression
  • Make it easier to enroll volunteers in clinical trials
  • Use the power of data to unlock breakthroughs
  • Catalyze promising therapy development


FSHD Drug Development pipeline

Our Purpose: Accelerate the development of treatments & discover a cure for FSHD.

Our Goal: Deliver disease-modifying therapies to our families by the year 2025.

Our Plan: Raise $15 million to for capital investment in:

  • Pre-clinical, pre-competitive infrastructure for clinical trials via an industry consortium, including a clinical trial network, patient registries, natural history studies, clinical outcome measures, biomarkers, imaging markers, and FDA guidance.
  • Venture philanthropy fund to invest in proof-of-concept and early clinical development of the most promising treatments.


Bridge to FDA approval

The chief obstacle that stands between us and this vision of a world free of the burdens imposed by FSH muscular dystrophy is the funding necessary to bring this dream to reality. Through your support, you have the power and ability to deliver treatments and a cure to nearly a million people affected by FSHD.

Because, at the end of the day, it is all about our families.

Interested in advancing the conversation? Please contact:

Mark Stone
President & CEO, FSH Society
(781) 301-7323; mark.stone@fshsociety.org