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Cystic fibrosis: triple therapy shows promising results

BMJ 2019; 367 doi: (Published 04 November 2019) Cite this as: BMJ 2019;367:l6347
  1. Elisabeth Mahase
  1. The BMJ

The triple therapy Trikafta (elexacaftor, tezacaftor, and ivacaftor) has the potential to lead to “transformative improvements in the lives of people with cystic fibrosis,” two phase III trials indicate.

The studies, funded by the drug company Vertex, looked at the efficacy and safety of Trikafta in patients with one copy of Phe508del—the most common gene mutation that causes cystic fibrosis—and patients with two Phe508del mutations.

Both trials reported positive results for the drug, which could benefit many patients, as up to 90% of all people with cystic fibrosis have at least one copy of the Phe508del mutation, and almost 50% have two copies.

The findings came after NHS England was able to …

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