Driving Breakthroughs for FSHD Treatments

No Time to Wait 

By Erin Saxon, FSHD Society

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Those living with FSHD face a harsh reality: this is a relentlessly progressive disease with no treatments to slow or stop it. That reality is on the cusp of change. With multiple therapies in clinical trials and more in the pipeline, we have entered a new era of possibility. The FSHD Society itself is also in a new era, growing in scope and leading global efforts to bring safe, effective therapies to market as quickly as possible. 

This effort is as ambitious as putting a person on the moon, and we’re positioned to make it happen.  

Our Therapeutic Accelerator is an initiative that unites researchers, regulators, pharmaceutical companies, clinicians, industry partners, and patients. Together, we are building the knowledge, infrastructure, and advocacy needed to overcome the unique challenges of FSHD drug development. This is our moonshot: turning scientific breakthroughs into treatments that reach those who need them as soon as possible. 

The FSHD Society is uniquely equipped to drive this work. Over many years, we have cultivated a worldwide network of scientists and clinicians who are dedicated to solving FSHD. We have partnered with patients and families to gather real-world data, making sure that treatments in development address lived experiences. 

This means that the FSHD Society is more than a participant in the drug development process. We are the central hub, ensuring that every effort contributes to the larger goal of delivering safe and effective therapies. 

The Therapeutic Accelerator moves progress forward on three fronts: 

Advancing science through collaboration.  

• Each year, leading scientists gather at our International Research Congress to share discoveries and spark new ideas.  

• The Industry Collaborative brings companies together to tackle common obstacles in clinical trial design, measurement tools, and data sharing.  

• Our research grants support innovative projects that address unanswered questions in FSHD biology. 

Building clinical research infrastructure.  

• The Clinical Trial Research Network connects expert sites across the globe to deliver high-quality trials.  

• The National Registry and FOCUS database collect vital patient information from completed studies to inform future work.  

• BetterLife FSHD empowers individuals to track their own health, match with trials, and contribute valuable data to the research community.  

• Through the Global FSHD Innovation Hub, we partner directly with biopharma companies to speed product development. 

Ensuring access for patients.  

• Progress only matters if treatments reach people. The FSHD Navigator connects individuals with trials, specialists, and community support.  

• Our FSHD 360 conferences bring education and resources to families while strengthening relationships with local clinicians.  

• Large-scale gatherings like FSHD Connect build visibility and unity.  

• Project Mercury, now a global initiative, develops strategies to address the complex regulatory and policy issues that determine how quickly therapies become available. 

Developing treatments for FSHD is no longer a distant dream. Multiple clinical trials are underway, and the pipeline is growing. We have crossed an important threshold where the science is ready, the community is organized, and the Society is working steadfastly in its role as the central driver of progress. The FSHD Therapeutic Accelerator is ensuring that the science does not stall and that those living with FSHD do not wait longer than necessary. 

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This article was originally published in the The FSHD Advocate, 2025 Issue 2. Read the latest issue here.