As a non-profit organization we strive to do everything we can to meet our mission of accelerating research for FSH.
Because of the contributions of our generous donors over the last decade, we have achieved unprecedented progress on our understanding of FSHD.
- Scientists have 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 massive experiments to look for compounds that interfere 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 established so we know, when we find a therapy, whether it will work or not.
You can read all about where your contributions went in our Annual Report.