The future is in powerful hands: YOURS

I have been impressed with the urgency of doing. Knowing is not enough. We must apply. Being willing is not enough. We must do.”―Leonardo da Vinci

Throughout 2018, we have made huge strides in the twin pillars of our mission—to accelerate therapies while empowering families.

Thanks to your steadfast support, we launched our chapter program to serve individuals and families across the United States. Following the adage to “think globally and act locally,” our chapters support our members in their own communities while aligning their talents and energies toward our shared goals. Now 14 chapters strong—and growing steadily—our program is raising an “army of activists” that will change our world.

To speed up the development of treatments, we have engaged with multiple pharmaceutical companies and researchers that are investigating potential treatments. We have charted a clear path for the next several years, identifying the challenges that must be surmounted to move the most promising therapies through the clinical and regulatory maze, as well as marshalling the resources necessary to overcome each roadblock.

The Time Is Now!

Drug development has reached a crossroads. Some companies that have promising drugs are delaying investment in their FSHD programs because they perceive a lack of accepted criteria to prove that a drug is effective and deserving of FDA approval. Researchers agree that the DUX4 gene is a key factor and suppressing it will likely halt the progression of FSHD, but without validated methods to measure this in a clinical trial setting, some companies are hesitant to continue working on FSHD. Interestingly, when we outlined our plan to solve these issues through an aggressive “trial readiness” research initiative, all of these companies were immediately encouraged.

Following the path of other successful, research-focused patient advocacy groups, the FSH Society is in a unique position to bring all of the stakeholders to the table to collaborate on solutions. By engaging in a creative partnership with industry, researchers, the FDA, and our families, we are designing and funding projects that will clear the path to FDA approval of FSHD therapies.

There is no time to waste! Clinical trial readiness projects like these need to be completed or well underway within the next three years. To succeed, the Society must commit financial resources for the three years that these projects will take.

The time to act is now. Your commitment will enable us to make targeted investments that will allow effective therapies to advance quickly through the approval process. As a member of our community dedicated to ensuring that our families have access to disease-modifying treatments by 2025, I hope this season that you will give—and give generously—to make this vision a reality.

We have a clear and certain direction, and the members of our Board of Directors have demonstrated confidence in our plan by pledging $400,000 and challenging all of our members to match their gift.  Please help us meet—and surpass—this goal by donating as generously as you can before December 31.

It’s going to take all of us to cross the finish line, but with your continued support, we will reach our shared goal―treatments for our families by 2025.

Working with you in passion and purpose,

Mark Stone


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