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  • Plenary Session I: Thursday, October 31, 2019

    "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. 

    Topics: Gene Editing, Health Outcomes, CFTR, Plenary
  • Plenary Session II: Friday, November 1, 2019

    "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.

    Topics: Health Outcomes, CFTR, Plenary
  • Plenary Session I: Thursday, October 18, 2018

    "Improving Outcomes of Infections in the Age of CFTR Modulators"

    Speaker: Lisa Saiman, M.D., M.P.H.

    In this session, Dr. Lisa Saiman will address the challenges of infections in people with CF and discuss why infections remain a major focus of the CF community, even in the era of effective CFTR modulators. She will provide an overview of CF infections, describe ongoing studies to increase our understanding of CF microorganisms and their optimal treatment, and discuss current strategies to develop new anti-infective agents to treat even the most difficult infections.

     

    Topics: Health Outcomes, Treatment, CFTR, Plenary, Clinical Research
  • Plenary Session II: Friday, October 19, 2018

    "Anti-inflammatories & Mucociliary Clearance Therapies in the Age of CFTR Modulators"

    Speaker: Felix Ratjen, M.D., Ph.D.

    This plenary will review the effect of CFTR dysfunction and CFTR restoration on both inflammation and mucociliary clearance in individuals with CF. Current and future therapeutic strategies to address inflammation and mucociliary clearance will be discussed, as well as how these strategies have potential to benefit both those patients using CFTR modulators and those not eligible for modulators.

    Topics: Health Outcomes, Treatment, CFTR, Plenary, Clinical Research
  • Plenary Session I: Thursday, November 2, 2017

    "Matching Medicines With Mutations"

    Speakers:   Michael P. Boyle, M.D., FCCP and Philip J. Thomas, Ph.D.

    Increasing numbers of people with CF now benefit from disease-altering small molecule therapies that target the basic defect in CFTR.  Use of these therapies has been extended recently using a combination of in vitro laboratory experiments to identify responsive mutations, and clinical trials to determine effect on clinical outcomes. This plenary will summarize how recent advances may allow in the near future over 90% of individuals with CF to benefit from therapies that target the basic defect in CFTR, including even those with the rarest disease causing mutations.

    Topics: Health Outcomes, Treatment, CFTR, Plenary, Clinical Research, Drug Development Pipeline
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