Last year, the FSHD Society launched an aggressive initiative to accelerate therapeutic development in FSHD. Because of our donors’ 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.
This year, we had planned a targeted strategy focused on four key areas:
• better, cheaper, and faster genetic testing;
• a blood biomarker test to indicate whether or not a drug is working;
• integration of global patient registries to gather robust natural history data;
• Voice of the Patient Forum to provide the FDA with FSHD patient-informed evaluation criteria.
These initiatives are essential to our goal of delivering therapies to our families by 2025. And despite the immense challenges of this year, we have persevered. We have to, because TIME = LIVES. Every day of delay in carrying out our work is a day that symptoms worsen and our loved ones suffer.
Your support literally keeps us going. Our Board of Directors, comprised almost entirely of individuals and family members who live with FSHD, has pledged $500,000 toward our year-end campaign and challenges you to match their generosity. The work we have begun and the progress we have achieved require that we commit additional funding and staff time to key projects and research initiatives. Between now and the end of 2020, we need to raise the financial resources necessary to advance these projects and ensure we can complete them. Please consider making a gift at this most critical moment. Thank you.