FSHD Connect: How the Conference Brings the FSHD Community Together

by Raymond Huml, MS, DVM, RAC-US 

---

As both the father of two children living with FSHD and a biopharmaceutical industry veteran with more than 30 years of experience, including senior leadership roles in rare disease, I was honored to present the keynote address at the FSHD Connect meeting in Chicago. My goal was to provide a fair-balanced but optimistic view of the current FSHD landscape. I remain more hopeful than ever that we will see a disease-modifying treatment for FSHD in my lifetime, but I also believe that hope is most powerful when it is grounded in science, realistic expectations, and a clear understanding of the work still ahead. 

FSHD Connect felt different from the research-focused IRC, even though many people attended both meetings. The IRC is centered on scientific exchange, clinical development, and research progress. Connect, by contrast, is where that progress becomes deeply personal. It is a place where individuals living with FSHD, caregivers, family members, clinicians, researchers, advocates, and industry professionals come together not only to learn, but also to listen to one another and build relationships. 

That sense of connection was especially meaningful to me as the co-founder, along with my daughter, of the FSHD Society’s North Carolina Chapter. I was delighted to meet Michael “Mike” King, who now leads the chapter. Mike was diagnosed with FSHD in 2024 and now serves as Chapter Director. He struck me immediately as outgoing, empathetic, and genuinely committed to supporting others. Meeting him reinforced how important local chapters are in helping people move from isolation to community. 

 

When my daughter was first diagnosed more than 20 years ago, there were far fewer resources available to families seeking clear, practical information about FSHD. The Muscular Dystrophy Association and the FSHD Society were among the few organizations where we could find disease-specific support, education, and community. In those early years, I became friends with Carol Perez, the mother of one of the FSHD Society’s co-founders, and with her son, who helped build the organization before passing the baton to Mark Stone. Those relationships helped shape my understanding of advocacy as something deeply personal, built one conversation and one family at a time. 

That history made FSHD Connect particularly powerful for me. Today, families arriving at a meeting like Connect have access to information, expertise, and peer support that simply did not exist in the same way two decades ago. They can hear from leading clinicians and researchers, ask practical questions, learn about symptom management and standards of care, and better understand how clinical trials and emerging therapies fit into the broader picture. For newly diagnosed individuals and families, that kind of access can be transformative. It can turn fear and uncertainty into knowledge, direction, and connection. 

Connect also serves caregivers in an important way. FSHD affects far more than muscles; it affects households, routines, mobility, independence, planning, and emotional resilience. Caregivers often carry questions that are different from those of the person with FSHD, and they need space to learn, share, and be supported as well. A conference like Connect validates that role and gives caregivers practical tools, a broader community, and a reminder that they are not navigating this journey alone. 

From an industry perspective, I believe Connect is equally valuable. Scientists, clinicians, researchers, and biopharmaceutical professionals can learn an enormous amount by being present in the community. Listening to people describe fatigue, pain, mobility changes, uncertainty, family impact, and the daily tradeoffs of living with FSHD adds context that no dataset can fully capture. It reminds us that drug development is not an abstract exercise. It is ultimately about improving lives in ways that matter to patients and families. 

That is why I would encourage more industry professionals, clinicians, and researchers to attend FSHD Connect, even if they are not personally affected by FSHD. The meeting provides a rare opportunity to see the full ecosystem in one place: people living with the disease, caregivers, advocacy leaders, clinical experts, scientists, and companies working toward new therapies. For those of us involved in research or drug development, it strengthens our sense of responsibility and helps ensure that our work remains anchored in the lived experience of the community we aim to serve. 

Most of all, FSHD Connect lives up to its name. It connects people to information, to resources, to one another, and to a future that feels more hopeful because it is being built collectively. I left Chicago grateful for the opportunity to contribute, encouraged by the strength of the community, and convinced that events like Connect are essential to preparing the FSHD community for the next era of care, research, and treatment.