Article
Caution advised in the use of CFTR modulator treatment for individuals harboring specific CFTR variants.
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Sept 2022
Raraigh Karen S, Lewis Michelle H, Collaco Joseph M, Corey Mary, Penland Christopher M, Stephenson Anne L, Rommens Johanna M, Castellani Carlo, Cutting Garry R
Abstract excerpt
In December 2020, the U.S. Food and Drug Administration (FDA) expanded the list of CFTR variants approved for treatment with CFTR modulators drugs from 39 to 183. Clinicians should be aware that individuals harboring certain variants approved for treatment may not respond to or benefit from this therapy. After review, the expert panel leading the CFTR2 project identified four categories of variants that may not...
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