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33rd Annual North American Cystic Fibrosis Conference


W17--NT-ET: *New & Emerging Therapies to Correct the Basic Defect


Nov 1, 2019 2:15pm ‐ Nov 1, 2019 3:50pm

Description

During this session, we will explore data supporting the development of therapies to address the basic defect in CF including gene editing, mRNA read-through, and CFTR protein modulation. The session will cover CFTR therapeutic development along the entire pathway from pre-clinical technical advancements and target identification to results of Phase I, II and III clinical trials.

Learning Objectives:

  • Identify appropriate molecular targets for CFTR-directed therapeutics.
  • Review safety of potential new CFTR-directed therapies.
  • Critically evaluate efficacy data from CFTR modulator clinical trials.

Note: Speaker Matthew Goddeeris' presentation was not recorded at his request.

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