Elexacaftor/tezacaftor/ivacaftor, a game-changer in cystic fibrosis: The Portuguese experience

Phase 3 trials of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) combination treatment in people with cystic fibrosis (CF) with ≥1 F508del-CFTR allele showed profound short-term effects on lung function, weight, and pulmonary exacerbations (PEx). The authors conducted a 12-month study to add evidenc...

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Veröffentlicht in:	Pulmonary pharmacology & therapeutics 2024-12, Vol.87, p.102328, Article 102328
Hauptverfasser:	Fragoso, E., Boaventura, R., Almeida, L., Amorim, A., Gamboa, F., Santos, A.S., Gonçalves, F., Cruz, C.M., Carreiro, A., Gonçalves, A.S., Teixeira, V., Azevedo, P.
Format:	Artikel
Sprache:	eng
Schlagworte:	Adolescent Adult Aminophenols - adverse effects Aminophenols - therapeutic use Benzodioxoles - adverse effects Benzodioxoles - therapeutic use Chloride Channel Agonists - adverse effects Chloride Channel Agonists - therapeutic use Cystic Fibrosis - drug therapy Cystic Fibrosis - genetics Cystic Fibrosis - physiopathology Cystic Fibrosis Transmembrane Conductance Regulator - genetics Drug Combinations Female Forced Expiratory Volume Humans Indoles - administration & dosage Indoles - adverse effects Indoles - pharmacology Indoles - therapeutic use Male Middle Aged Portugal Pyrazoles - adverse effects Pyrazoles - therapeutic use Pyridines - adverse effects Pyridines - therapeutic use Pyrrolidines - adverse effects Pyrrolidines - therapeutic use Quinolones - administration & dosage Quinolones - adverse effects Quinolones - therapeutic use Treatment Outcome Young Adult
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