Care for patients with cystic fibrosis has undergone transformative changes over the past decade and serves as an example of how an understanding of the functional consequences of a genetic disease ...
A 24-week phase 3 trial has found that once-daily vanzacaftor-tezacaftor-deutivacaftor is generally safe and well tolerated in children with cystic fibrosis aged 6-11 years. The treatment maintains ...
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