Meeting in Munich: Why Bringing People Together Brings Us Closer to Answers

This past fall, my wife Cecilia and I sat across from a VCP family in a Munich café, watching as years of isolation melted into recognition and relief. They had traveled from Austria specifically to meet us, representing the first time they had ever met another family affected by VCP disease. Despite the language barriers, we understood each other perfectly. The diagnostic odyssey. The uncertainty. The determination to find answers. The hope that connection brings. We've always known that VCP disease goes far beyond impacting only the families we personally know, but this moment—thousands of miles from our home—served as a powerful reminder of the disease's broad impact and the enormous opportunity we have to bring people together and provide hope.

This encounter represented everything Cure VCP Disease exists to do: bring together VCP patients and families from around the world into a community that provides support, education, awareness, and drives research toward therapies. But this week in Munich would prove that the power of connection extends far beyond patient-to-patient relationships.

I was selected for the AstraZeneca Young Health Impact Fellowship program to attend the One Young World Summit in Munich, chosen from a competitive pool of young health leaders worldwide. Cecilia, also a rare disease advocate, traveled to Germany with me.

What began as an incredible honor became a week of profound conversations that demonstrated how collaboration across every boundary—geographic, organizational, linguistic—is essential to advancing rare disease treatment development.

Inside Alexion: When Patient Voices Meet Drug Development

Before the conference began, I participated in a panel discussion at Alexion headquarters in front of leadership and employees from their drug availability department. Addressing professionals who dedicate their careers to developing and delivering treatments for rare diseases, I explained what patient advocacy organizations uniquely contribute: we connect all the stakeholders required to advance therapies—researchers, clinicians, patients, families, and industry partners.

I also spoke about empowering young adults at risk of rare genetic diseases. When young people understand they can impact not only their own disease trajectory but the broader rare disease landscape through research participation, they move from fear to empowerment and control. This transformation increases research engagement, strengthens datasets, and accelerates the path toward treatments. The audience's response was encouraging—they wanted to understand the rare disease perspective from those of us living it, and that openness created space for genuine dialogue.

Walking through Alexion's facilities together, we witnessed the immense effort that goes into not only developing treatments but ensuring patients can access them. These decisions are carried out by talented, passionate people who genuinely care about solving these problems from the industry side.

The Question That Connects Us All

Throughout the week, nearly every conversation—whether with the Austrian family sharing their diagnostic journey, with AstraZeneca's CFO discussing investment factors, or with Alexion teams working on drug access—circled back to the same fundamental question: How do you make treatment development for rare diseases scientifically and economically viable?

In our conversation with AstraZeneca's CFO, we discussed the critical factors that enable pharmaceutical companies to invest in rare disease therapeutics: validated biomarkers, natural history studies, and stakeholder collaboration to accelerate development timelines. We engaged openly with the difficult questions at the heart of rare disease drug development.

Sometimes, as patient advocates, we can feel like we're fighting alone. But this week revealed allies working from every side: the Austrian family seeking to connect with others who understand their journey, the AstraZeneca and Alexion teams wrestling with scientific and regulatory complexity, and young health leaders from around the world tackling different diseases but facing parallel challenges. The investments that companies like AstraZeneca make in young health leaders and in creating spaces where patient voices inform industry strategy demonstrate a commitment that extends beyond individual drug development programs.

The Next Generation Takes the Lead

Throughout the conference, one truth became increasingly clear: solving the challenge of making rare disease drug development scientifically and economically viable will require the next generation to be actively engaged. The willingness to engage, to contribute lived experience, to participate in research—this is what young adults can bring to the table to make a meaningful impact. Young adults aren't simply the future beneficiaries of treatments; they are essential partners in creating the solutions that will get us to a treatment. 

Looking Ahead: The 2026 Summit

The 2026 Cure VCP Disease International Scientific and Community Summit will feature a dedicated session on young adult health in rare disease. Building on the conversations started in Munich, this session will connect young adults at risk, researchers, and industry partners to explore how we can better support and engage the next generation of rare disease advocates and research participants. It's an opportunity to put into practice what we witnessed in Munich: creating space for young adults to lead, to problem-solve, and to reshape how we approach treatment development.

Moving Forward Together

We left Munich more hopeful than when we arrived—not because the challenges became smaller, but because the network of people committed to solving them became clearer and more connected.

We didn't solve the question of how to make rare disease treatment development viable. But we came together to work toward the answer—families, advocates, researchers, industry leaders—and in that collaboration, we're making real progress. Every VCP family who joins our registry, every researcher who shares data openly, every industry partner who creates space for patient voices, every young adult who chooses engagement over fear—each is a vital part of the solution.

The question remains before us. But after Munich, we're convinced we're working toward it together—and that collaboration is what will ultimately lead us to the answer.