In January of 2014 we held our inaugural PAAC Leadership Workshop in Washington,DC. The day included engagement with federal policy makers, congressional staff, and research leaders. At the end of the day, a long-time colleague of mine from the FDA who had spent the entire day with us pulled me aside and said, “Wow. These men are brilliant. They have such…

Remember that hot June day in 2015 when hundreds of Duchenne community members rallied together at the U.S. Capitol for PPMD’s EndDuchenne rally? That same day, our longtime Congressional champions Senators Roger Wicker (R-MS) and Amy Klobuchar (D-MN) introduced the Patient Focused Impact Assessment Act (PFIA, S. 1597) — and our 200+ PPMD advocates…