Clinical response to lumacaftor-ivacaftor in patients with cystic fibrosis according to baseline lung function.

Fiche publication


Date publication

juin 2020

Journal

Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society

Auteurs

Membres identifiés du Cancéropôle Est :
Pr ABELY Michel


Tous les auteurs :
Burgel PR, Durieu I, Chiron R, Mely L, Prevotat A, Murris-Espin M, Porzio M, Abely M, Reix P, Marguet C, Macey J, Sermet-Gaudelus I, Corvol H, Bui S, Biouhee T, Hubert D, Munck A, Lemonnier L, Dehillotte C, Silva JD, Paillasseur JL, Martin C,

Résumé

Phase 3 trials have demonstrated the safety and efficacy of lumacaftor-ivacaftor (LUMA-IVA) in patients with cystic fibrosis (CF) homozygous for the Phe508del CFTR mutation and percent predicted forced expiratory volume in 1 s (ppFEV) between 40 and 90. Marketing authorizations have been granted for patients at all levels of ppFEV.

Référence

J. Cyst. Fibros.. 2020 Jun 23;: