Project 2025

Dear Friends,

At the FSH Society, our moonshot is to get to a treatment for FSHD―an intervention that will slow or halt the muscle deterioration. We have set a target year, 2025, a date our scientific advisors agree is realistic to get the first disease-modifying drug to our families. It is an ambitious goal, but we will succeed as long as we have your support, our shared vision, and—above all—the team that will get us there.

Our team—insistent patients and families, determined advocates, compassionate clinicians, and focused researchers—they are the engineers and astronauts who will land us on our moon. Individuals like Ryan Wuebbles, who doesn’t simply dream of a cure for himself, but has staked his life and career on making that dream a reality.

Growing up in California and Illinois, Ryan was an athletic kid, passionate about basketball, baseball, and soccer. One day, at age 12, he suddenly could not complete a sit-up. By age 16, his running had slowed, and he knew something was “weird.” Four years later, in college, he was diagnosed with FSHD.

It was devastating to lose the ability to participate in the sports he loved, but feeling he needed to take positive action, Ryan attended his first FSH Society meeting in 2002. “It was intimidating” to walk into a room full of people who shared his diagnosis and see what his future might hold, “but it helped me define what I want to do with my life,” he recalled.

Ryan decided to pursue a PhD in biology at the University of Illinois. Because FSHD had been linked to “repeat units” in the genome—a signature associated with epigenetics, inheritable biological mechanisms that change the expression of our genes—Ryan found his way to Peter Jones, who had just joined the Illinois faculty.

“Peter was looking for students. He worked on epigenetics, and his understanding was far greater than anyone else’s at Illinois at the time,” Ryan explained. Ryan joined the lab and persuaded Jones to take a look at the disease that had upended his life.

The rest is history. Peter and his wife Takako have since risen to the front ranks of FSHD research, with groundbreaking discoveries about epigenetic regulators affecting the severity of symptoms. They have created a genetically engineered mouse model of the disease and, with Charis Himeda, pioneered a CRISPR-based gene-editing method to suppress the gene implicated in FSHD.

Ryan earned his doctorate in 2009, got married, and joined the University of Nevada, Reno, medical school. There, he teamed up with Professor Dean Burkin and helped found Strykagen, a biotech company working on treatments for muscle diseases, including FSHD and Duchenne muscular dystrophy.

Over the years, FSHD has chipped away at Ryan’s strength, and he has transitioned to using a scooter, but it has not diminished his determination or optimism. He and his wife now have two sons, ages three and six. “Though FSHD has stripped my ability to play most sports,” he said, “the confidence and work ethic I learned through sports has been channeled into my pursuit of a career in science and my work to find treatments for muscle diseases.”

Ryan Wuebbles personifies the heart of the FSHD community—all of us pulling together to face the challenges and dedicate ourselves to making sure that all who live with FSH muscular dystrophy can reach their full potential. No one should have to live with the pain and unrelenting loss of ability that this condition brings.

We are focused on the two components that will propel us to success. First, we are directing our funding to research and infrastructure that will greatly speed up the development of treatments. And second, we are enlarging and activating our community, and empowering our stakeholders to raise awareness, engage, and advocate for the resources we require. These are the fuels that will power our rocket engines.

Your loyal support has helped us reach this tipping point. Together, we have come very far. This year, with your continued support, we will go so much further! We will:

• launch a major FSHD therapeutics initiative designed to accelerate the pace and volume of drug development, getting disease-modifying treatments to our families faster;

• equip and empower you to become an effective “army of activists,” because the determination of a community of patients and families is the only thing that has ever made a difference in the outcome of drug discovery and development.

We expect 2018 to be a transformational year for the FSHD community. This is why a group of visionary benefactors has pledged to match up to $100,000 in gifts made by May 31, 2018. By donating to our spring campaign, you will give us the fuel to reach our moon—a treatment to slow or halt FSHD—by the year 2025. Thank you for being on our team!

Serving together,

Mark Stone
PRESIDENT & CEO