10/09/2026
💙 Small voices can make a BIG impact!
Honored to join Michele Oshman at this year’s Rare Trials Summit to share our family’s journey and highlight how Maggie has used her voice to advocate for the Rare Pediatric Disease Priority Review Voucher (PPRV) Program! 💪🏼
For children like Peter and Maggie living with rare diseases like Limb-Girdle Muscular Dystrophy Type 2C (LGMD2C), programs like the PPRV are critical to driving research, encouraging innovation, and bringing life-changing treatments to children who simply cannot afford to wait.
We are so proud of Maggie for continuing to use her voice to advocate not only for herself and her brother, but for children everywhere living with rare diseases. 💙
A heartfelt THANK YOU to for giving our family the opportunity to share our story, amplify the voices of the rare disease community, and continue advocating for the programs that give our children hope.
Because rare doesn’t mean forgotten. And our children can’t wait. 💙
RareDisease GeneTherapy ChildrenCantWait NoChildLeftBehind
One afternoon in 2024, the BIO Federal Government Affairs team received a call from Hill staff who said, “We just met the most amazing family. They came down from Boston. They have a story to share, and we think you guys need to know them.”
Joe and Courtney Dion came to Washington to share their story: Peter, age 10, and Maggie, age 6, were born with limb-girdle muscular dystrophy (LGMD-R5, formerly known as LGMD-2C), a rare disease that would eventually rob the children of their ability to walk, move independently, and breathe on their own.
At the time, there was little hope on the horizon.
“Our first conversation with the Dions was incredibly powerful—it became clear that they had done their homework. They identified a company with a promising gene therapy, but which could not progress to the clinic without significant investment,” explains Michele Oshman, BIO’s Chief Patient Advocate. “They also understood that investment could very well depend on the reauthorization of a little-known, but incredibly powerful federal program called the pediat
