Article
Ivacaftor improves lung disease in patients with advanced CF carrying CFTR mutations that confer residual function.
Respiratory medicine - 1 Sept 2020
Salvatore Donatello, Terlizzi Vito, Francalanci Michela, Taccetti Giovanni, Messore Barbara, Biglia Carlotta, Pisi Giovanna, Calderazzo Maria Adelaide, Caloiero Mimma, Pizzamiglio Giovanna, Majo Fabio, Cresta Federico, Leonetti Giuseppina, De Venuto Domenica
Abstract excerpt
BACKGROUND: Ivacaftor is an innovative treatment for CF. Ivacaftor monotherapy in a phase III trial for patients with F508del and a residual function (RF) mutation showed improvement in lung function. We evaluated the effectiveness and safety of ivacaftor in patients with severe CF carrying RF mutations. METHODS: Data were collected from Italian CF centers with patients enrolled in an ivacaftor compassionate use...
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