Explore the NACFC Mobile App Menu
  • 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
  • Plenary Session II: Friday, November 3, 2017

    "Lung Transplantation: Challenges & Opportunities for Advanced CF Lung Disease"

    Speakers:  Joseph Pilewski, M.D.

    Plenary 2 will review the progression of CF lung disease and the option of lung transplant to treat advanced disease. The presentation will highlight the challenges associated with each phase of the transplant journey, including the referral to a transplant center, waiting for a transplant, undergoing the transplant procedure, and the care requirements and life after transplant.  

    Topics: Clinical Trials, Plenary, Clinical Research, Drug Development Pipeline
  • Plenary Session III: Saturday, November 4, 2017

    "Partnering With the Community"

    Speaker: Drucy Borowitz, M.D. and Mary B. Dwight

    This plenary session will focus on ways people with CF and their families make a difference by partnering with the larger community as advocates, by sharing their expertise on living life with CF with their care center teams, and by supporting with each other through virtual events and peer mentoring.  

    Topics: Health Outcomes, Treatment, Care Model, Observational Research, Plenary
  • Plenary Session I: Thursday, October 27, 2016

    "A Cure for All: Leaving No One Behind"

    Speakers: Mitchell Drumm, Ph.D. and Eric Sorscher, M.D.

    This session describes recent advances toward treatment of cystic fibrosis and the pipeline for new and emerging therapies.

    Topics: Gene Editing, Treatment, Personalized Medicine, Plenary, Drug Development Pipeline
  • Plenary Session II: Friday, October 28, 2016

    "Clinical Research: A Worldwide CF Community Effort"

    Speakers: Christiane De Boeck, M.D., Ph.D. and Patrick Flume, M.D.

    The cystic fibrosis clinical trial pipeline is at present very busy -- and this is great news. This session explains how worldwide collaboration and streamlining can optimize the efficiency in the clinical trial arena.

    Topics: Clinical Trials, Plenary, Clinical Research
  • Plenary Session III: Saturday, October 29, 2016

    "You Can Observe A Lot by Just Watching: Lessons from The CFF Patient Registry"

    Speaker: Wayne Morgan, M.D.

    This plenary session reviews the role of observational research in improving our understanding of cystic fibrosis, the care of patients with CF, and opportunities to improve this care, highlighting the role of the CF Foundation Patient Registry.

    Topics: Patient Registry, Observational Research, Plenary
Showing 1–6 of 9 results