Three Years. Ten Countries. One Shared Mission…What We Built Together 

Reflections from Mark Stone  

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Three years ago, Project Mercury began with a simple but urgent realization: promising therapies for FSHD were advancing faster than the world’s ability to deliver them to patients. 

The science was accelerating. Clinical pipelines were growing. Hope was becoming tangible for families who had waited generations. But beneath that momentum sat a hard truth. The global system was not ready. 

There were too few clinical trial sites. Too few patient registries. Limited coordination between countries. Fragmented evidence frameworks. Vast differences in healthcare preparedness. And perhaps most importantly, there was no existing structure capable of solving these challenges at the scale required.  

So, in 2023, we decided to build something different. 

Not another isolated initiative. Not another organization protecting its own lane. But a patient-led, globally coordinated movement built around a shared mission: ensuring that when therapies arrive, patients everywhere are ready to receive them. 

Today, as we reflect on the first three years of Project Mercury, I am deeply proud not only of what we accomplished, but of how we accomplished it together. 

From Concept to Global Movement 

What began as an ambitious idea is now an operational reality spanning ten countries: Australia, Brazil, Canada, France, Germany, Italy, the Netherlands, Spain, the United Kingdom, and the United States. 

Across those countries, we built Country Working Groups that brought together patient advocates, clinicians, researchers, industry leaders, health economists, regulators, and subject matter experts. Coordinating the initiative, a Global Task Force emerged. Not as a governing body dictating direction, but as a collaborative engine connecting expertise, experience, and shared purpose across borders. 

What makes me most proud is that this was never simply a technical exercise. Project Mercury was built by purpose-driven people. 

By volunteers who stayed up late joining meetings across impossible time zones. By advocates who balanced this work with jobs, caregiving, and their own progression with FSHD. By organizational leaders donating not only institutional support, but their time, creativity, and emotional energy. By countless numbers of individuals who financially supported this work. And by clinicians and researchers willing to challenge long-standing assumptions and rethink what global collaboration could look like.  

And yes, by arguments. 

Real collaboration is not always smooth. We debated priorities. We wrestled with differences in healthcare systems, resources, and philosophies. We confronted difficult questions about equity, sustainability, and responsibility. But those disagreements were not weaknesses. They were signs that people cared deeply enough to fight for what mattered. 

And through those conversations, something remarkable happened: a true global community began to mature. 

The Progress We Can Measure 

The results of the first three years are substantial. 

Together, Project Mercury countries launched or expanded FSHD registries across six countries, ultimately surpassing 10,000 patients enrolled globally. We more than doubled the number of qualified clinical trial sites, growing from 25 sites in 2023 to 53 sites in 2025. 

We developed and published a toolkit for new FSHD clinical trial sites. We convened international workshops to modernize trial readiness and operations. We initiated global consensus efforts around clinical monitoring and real-world data standards. We began building disease progression and health technology assessment frameworks designed to help payers and regulators better understand the lived reality of FSHD. 

But perhaps most importantly, we shifted the conversation itself. 

Historically, patient access work in rare disease has often been reactive and product-specific, driven primarily by manufacturers after therapies approach approval. Project Mercury challenged that paradigm by pursuing advocacy-led, disease-level preparedness years before therapies reach patients. That shift matters. 

Because access delays are not abstract policy problems. They are years of lost mobility. Lost independence. Lost opportunities. Lost time families never get back. 

Preparing healthcare systems before approval, not after, may ultimately become one of Project Mercury’s most important contributions. 

Building Beyond Silos 

One of the most encouraging developments over these three years has been watching local and regional leadership strengthen across the world. 

Country Working Groups evolved from early coordinating bodies into increasingly sophisticated national ecosystems with their own sustainability models, advocacy capacity, educational programming, and strategic priorities. Countries learned from one another instead of duplicating effort. Templates, systems, and resources were shared openly. Success in one region became acceleration for another. 

At the regional level, the emergence and growth of FSHD Europe has been especially meaningful. Europe demonstrated what coordinated regional collaboration could look like in practice – bringing together diverse healthcare systems, advocacy organizations, and clinical networks under a shared vision for preparedness and patient access. 

At the same time, our partnership with TREAT-NMD through the Innovative Health Initiative-funded PaLaDIn project opened an entirely new chapter for Project Mercury. Through PaLaDIn, we are helping demonstrate how patient advocacy organizations can lead cross-sector collaboration, leverage real-world evidence, and contribute meaningfully to health technology assessment readiness in rare disease. 

This is larger than FSHD now.  What we are building has implications far beyond our own community. 

The Human Infrastructure 

When people read reports, they often focus on metrics; patient counts, trial sites, publications, frameworks, grants…. Those things matter deeply. But the true infrastructure we built over these three years cannot be measured entirely in numbers. It lives in relationships. 

It lives in the trust developed between advocates and clinicians who once barely knew one another. In the confidence of smaller patient organizations that discovered they had a seat at the global table. In the willingness of experts from industry, academia, and advocacy to work side-by-side instead of in parallel. 

It lives in the countless moments nobody sees: 

• Volunteers translating materials late at night. 
• Advocates mentoring newly formed groups in other countries. 
• Families sharing painful experiences so future patients may face fewer barriers. 
• Researchers listening more carefully to patient voices. 
• Leaders learning when to compromise and when to stand firm. 

That human infrastructure is what makes Project Mercury durable. And it is what gives me confidence that the work will outlast any single report, chairperson, or organization. 

The Road Ahead 

We are now entering perhaps the most critical phase of all. The first generation of FSHD therapies is moving into phase three clinical trials. The transition from scientific possibility to healthcare reality is no longer theoretical and it is approaching rapidly. 

The work ahead will require even more from all of us: 

• Preparing healthcare systems for treatment delivery. 
• Expanding clinical capacity. 
• Supporting equitable access. 
• Building stronger evidence frameworks. 
• Ensuring underrepresented patient populations are not left behind. 
• Transitioning Project Mercury from a time-limited initiative into a sustainable global platform within the 31+ country World FSHD Alliance ecosystem. 

But we are no longer starting from zero. Three years ago, global preparedness for FSHD therapies barely existed. Today, there is a coordinated international movement ready to help carry this work forward. 

And that belongs to every person who contributed – every patient advocate, every Country Working Group member, every Global Task Force participant, every organizational leader, every clinician, every researcher, every partner, and every volunteer who believed this community could achieve more together than any of us could alone. 

Thank you for helping build Project Mercury. The journey continues, stronger than ever.