EPP

S24--Bridging the Gaps: Extrapulmonary Complications Not Addressed by HEMT.

Chair(s)

• 

  Kim McBennett, MD, PhD

  Case Western Reserve University
• 

  Leah Reznikov, PhD

  University of Florida

Cystic Fibrosis is an autosomal recessive disease caused by mutation of the gene coding for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The lack of functioning protein leads to dysfunction of multiple organ systems, with the primary source of morbidity and mortality being the pulmonary system. In the past decade, the advent of highly effective modulator therapies (HEMT) has meant that we can augment the production, trafficking, and function of CFTR with oral therapies. Highly effective modulators have improved pulmonary symptoms and decreased the rate pulmonary decline for many people with a qualifying genetic mutation. However, it’s become clear that many of the morbidities attributed to cystic fibrosis are either not dependent on a functioning CFTR, or are irreparable after a particular stage of development. These morbidities, and their lack of a clear response to highly effective modulators, will define the future of cystic fibrosis clinical care and research.

Presentations:

• 2:30 PM – 4:30 PM ET

  S24.1- Men’s Health in Modern Era of Cystic Fibrosis
  

  Speaker: Traci M. Kazmerski, MD, MS (she/her/hers) – University of Pittsburgh School of Medicine
• 2:30 PM – 4:30 PM ET

  S24.2- Cystic Fibrosis Liver Disease in the Post-modulator Era
  

  Speaker: Michael R. Narkewicz, MD (he/him/his) – University of Colorado SOM and Children's Hospital Colorado
• 2:30 PM – 4:30 PM ET

  S24.3- Current Landscape of cystic fibrosis-related Diabetes & Modulator Therapy
  

  Speaker: Bernadette Prentice, MBBS PhD (she/her/hers) – Sydney Children's Hospital
• 2:30 PM – 4:30 PM ET

  S24.4- Mental Health Well-being & Associations with Lung Outcomes
  

  Speaker: Kevin Psoter, PhD – Johns Hopkins University