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...
Patient Engagement Does Not End With Understanding Patient Needs When organizations think about patient engagement, the conversation often focuses on insights. What are patients experiencing? What barriers are they facing?...
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...
Today brings exciting news for the rare disease community, born from immense effort, patient advocacy, and a refusal to back down. In a historic move, Metachromatic Leukodystrophy (MLD) and Duchenne...
