Archives

"Emerging Technologies for CFTR Restoration in All People With CF"

Speaker: Marie Egan, M.D.

Genetic therapies, including RNA therapies, gene therapies, and gene editing, hold the key to a cure for cystic fibrosis and are moving toward the clinical realm at an unexpected pace. Moving from the promise of these technologies to the reality of effective therapies for the underlying cause of the disease for all people with CF and a cure for CF will require time, tenacity, and vision.

Marie Egan, MD, from the Yale School of Medicine, reviews the power of genetic therapies, outlines the obstacles and challenges that lie ahead, and discusses what success looks like as we move toward therapies that address the underlying defect for all people with CF and a cure for cystic fibrosis. 

"Entering the Era of Highly Effective Modulator Therapy"

Speaker: Jane C. Davies, M.D.

In this session, Professor Jane Davies from the National Heart & Lung Institute, Imperial College in London, describes the progress and the promise of highly effective CFTR modulator therapy for people with CF. She highlights the long-term benefits such therapies have had on the relatively small proportion of the CF community for whom a highly effective modulator has been available for several years -- those with the gating mutation G551D who are on ivacaftor -- and uses this evidence to describe the impact we might expect from a triple-combination drug in a larger population. The additional opportunities posed by introducing CFTR functional restoration in early life is illustrated with pre-school and infant data.

"Evolution of CF Care: Innovation & Impact"

Speaker: Peter Mogayzel, M.D., Ph.D.

In this session, Dr. Peter Mogayzel walks us through past challenges and how our care model has met those challenges and built upon what we've learned to prepare for the future. As we enter a new era of therapeutics, now is the moment to consider what should shape how CF care evolves to meet the changing needs of people with CF and their families. Following his presentation, Sue Sullivan, RN, from Oregon Health & Science University; Terri Laguna, MD, MSCS, from Ann and Robert H. Lurie Children's Hospital of Chicago; and Cindy Brown, MD, of Indiana University, join Dr. Mogayzel for a panel discussion about the factors that should influence how CF clinical care evolves to support the needs of everyone with CF.