Cure HSPB8 at World Orphan Drug Congress USA 2025: Accelerating the Path to a Cure
We’re still energized after an incredible few days at the World Orphan Drug Congress USA 2025, held April 22–24 in Boston! This dynamic gathering brought together over 2,000 stakeholders across the rare disease ecosystem—scientists, regulators, biotech innovators, investors, and patient advocates—all united by one mission: to transform the future for rare disease patients.
Why We Attended
Attending WODC was an important opportunity for us to:
Share our latest research progress
Raise awareness about HSPB8 Myopathy
Build collaborations with scientists, regulators, and industry partners
Learn about cutting-edge advancements and policy changes shaping the future of rare disease drug development
Our team—President and Founder Todd King, Program Director Dr Ania Kordala, and Science Advisor Matt McLeod—attended and presented our poster on Cure HSPB8’s mission, strategy, and progress, helping to amplify our voice within the broader rare disease community.
What the Conference Is About
The World Orphan Drug Congress USA is the premier event focused on rare disease and orphan drug innovation. With dozens of expert-led sessions and unparalleled networking opportunities, it covers everything from regulatory science and gene therapy development to commercialization and patient access.
Key Takeaways for Cure HSPB8
Here are five takeaways that especially resonated with our mission:
🔹 Regulatory momentum is building: The FDA is demonstrating increased flexibility in how it approaches ultrarare conditions like HSPB8 Myopathy.
🔹 New guidelines are emerging: Regulatory agencies are beginning to create tailored frameworks—not just for antisense oligonucleotides, but for the broader ultrarare space.
🔹 Traditional trial models don’t fit: Standard clinical trial designs simply don’t work for diseases with very small patient populations. Innovation is critical.
🔹 Adaptive trial designs are essential: Creative approaches that allow real-time learning and adjustment are urgently needed to produce meaningful evidence with fewer patients.
🔹 Global collaboration is non-negotiable: To gather enough data, overcome geographic limitations, and accelerate progress, working together across borders is a must.
Our Call to Action
At the current pace, it could take more than 200 years to develop treatments for all known rare diseases. We cannot afford that timeline. Every day we wait costs lives.
At Cure HSPB8, we’re committed to moving faster—through collaboration, innovation, and relentless advocacy. This conference reinforced what we already believe: No rare disease should be left behind.
Thank You
To everyone who stopped by our poster, shared ideas, or offered support—thank you. We’re grateful to be part of such a vibrant and forward-thinking community. Let’s keep pushing the boundaries together.
💙 The Cure HSPB8 Team
