Getting to a treatment or cure for rare diseases means engaging in scientific research. But you want to be sure that the scientific research moves toward a therapy rather than repeating old findings or pursuing an avenue of research with little promise. How can leaders of rare disease patient advocacy organizations assess the research landscape and accelerate research and drug development? One approach highlighted by many as a best practice is bringing on board a Chief Science Officer (CSO) to sort through the scientific information and provide dedicated, targeted guidance. Hear from rare disease leaders about how a CSO can transform your organization’s research efforts.
Speaker: Molly White, Chief Executive Officer, Myotonic Dystrophy Foundation
Speaker: John Porter, PhD, Neuromuscular Disease Consultant
Day 1, Track 3