Remembering Scott

The day after the legislative conference, 800 rare disease advocates from across the U.S. descended on Capitol Hill to meet with our Congressional representatives' offices. It is always moving to hear the stories of patients fighting for years to get to the correct diagnosis or the right treatment, or any treatment at all. Half of all rare disease patients are children, and it was heartbreaking and inspiring to hear stories from the parents or the children themselves, battling for their lives with grit and eloquence.          Meeting with Rep. Sarah Elfreth's office This year I joined the Maryland delegation to meet with the legislative staff for Rep. Sarah Elfreth, Rep. Glenn Ivey, and Sen. Angela Alsobrooks. I ended up leading two of the meetings, and our asks included: 1) Support funding for FDA's newly established Rare Disease Innovation Hub to connect experts and industry and accelerate rare disease treatment development and approvals 2) Support the Access...

Five weeks out from brain surgery, I wasn't sure if I would be able to attend Rare Disease Week this year. My energy level is not yet back to 100%. Because this is a cause that is close to my heart, I decided to attend but prioritized my time and energy on the legislative conference and meeting with my representatives' offices, and I'm glad I did. It was wonderful catching up with my fellow rare disease advocates, and Rare Disease Week feels like a reunion with friends, especially with my amazing Pheo Para Alliance team. Phearless Pheo Para Advocates Last year, the vibe at the rare disease legislative conference felt uncertain but determined. We were facing cuts to rare disease research and Medicaid, and key pieces of rare disease legislation were on the brink of being passed but then pulled at the last minute due to political crossfire. This year, we had several major victories to celebrate. Nearly all of our recent rare disease legislative asks were passed and signed in...