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. 2020 Jan 23;180(2):211.
doi: 10.1016/j.cell.2019.12.032.

A Therapy for Most with Cystic Fibrosis

Christine E Bear  1
Affiliations
Free article

A Therapy for Most with Cystic Fibrosis

Christine E Bear. Cell. .
Free article

Abstract

TRIKAFTA is the third drug approved by the FDA that rescues defects caused by the major mutation F508del. It is superior to its predecessors that were approved for patients who are homozygous for F508del because TRIKAFTA is also effective in CF patients who harbor only one copy of this mutation.

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