In policy meetings and committee rooms across the country, newborn screening conditions are regularly scored, debated, and weighed. Leaders evaluate analytical validity, treatment efficacy, and system capacity. But every so...
Rare Disease Recruitment Is Not a Volume Problem Many clinical trial recruitment strategies are built around reach. More awareness. More advertising. More referrals. More outreach. In common disease states, increasing...
Living with a rare disease is a challenge. Experiencing a life changing injury is a challenge. Experiencing both was life-altering. When One Diagnosis Becomes Two Life-Changing Challenges I was walking...
Living with Duchenne and the Importance of Clinical Trials I am 24 years old, living with Duchenne Muscular Dystrophy, and advocating for accessible air travel. Duchenne is a muscle-wasting disease...
Paving the Potholes in Advocacy We’ve all been there. You’re navigating a complex system—whether it’s healthcare, education, or government—and you hit a wall. You find yourself asking: That frustration is...
I Wrote a Book. Actually, I have now written two—a fact that still surprises me daily. If you had told me twelve years ago that I would be an author,...
Independence Shapes Everyday Life Independence is not a luxury – It’s a necessity, and it’s extremely important to me. For me, independence means being able to do things with little to no help from...
A Rare Diagnosis My husband and I both agree that Friday, February 13, 2015, was the worst day of our lives. We had endured a six-week diagnostic odyssey for our...
