Our Impact & Finances
As a non-profit organization we strive to do everything we can to meet our mission of accelerating research for FSHD.
Because of the investments of our generous supporters over the last 15 years, we have achieved unprecedented progress on our understanding of FSHD (see also Advancing Discoveries).
- Scientists have now reached a consensus on the root cause of FSHD and have identified two molecular targets for intervening with the disease.
- Platforms have been developed for lead compound identification and scientists are now performing experiments to look for compounds that alleviate with the disease. Other strategies, including viral gene therapy, are also showing great promise.
- Preclinical models of the disease have been developed, and are being used to test lead compounds and strategies.
- Clinical trial endpoints are being developed and standardized so that we can assess the efficacy of proposed therapies.
You can read all about where your contributions went in our Annual Report.