02/03/2025
Our NMRL team recently attended the 2025 Duchenne Healthcare Professionals Summit! This event, hosted by , brought together over 360 healthcare professionals, researchers, industry leaders, and international representatives to discuss the latest advancements in dystrophinopathy care and research.
Key highlights included insights from gene therapy developments, updates on approved therapies, and the addition of Duchenne to newborn screening panels in four states. To close the session, Dr. McDonald delivered a presentation on the “why’s” of clinical trial design.
For more information on duchenne and how you can advocate for our families and research, check out PPMD’s website!