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33rd Annual North American Cystic Fibrosis Conference


S06--NT-APP&D: Where Mechanism Meets Therapy Beyond CFTR


Oct 31, 2019 2:00pm ‐ Oct 31, 2019 3:45pm

Description

This session will focus on the mechanistic basis for the development of novel, non-CFTR directed small molecule therapies that may improve mucociliary clearance in CF.

Learning Objectives:

  • Discuss the mechanistic basis of non-CFTR-mediated anion (Chloride and Bicarbonate) transport in the CF airway, and how small molecules directed at alternate anion transporters may bypass mutant CFTR in the CF airway.
  • Discuss the mechanistic basis of hypothesized defects in sodium transport in the CF airway, and how targeting this defect with small molecules may improve the pathophysiology of the CF airway.
  • Discuss the mechanistic basis of distorted mucociliary clearance in the CF airway in the context of how utilization of osmotic and mucolytic interventions may lead to improvements.

Note: Speaker Ronald Rubenstein's presentation was not recorded at his request.

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