Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study

Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study

Ivacaftor is generally safe and effective in patients aged 2 years and older who have cystic fibrosis and specific CFTR mutations. We assessed its use in children aged 12 to

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Bibliographische Detailangaben
Veröffentlicht in:The lancet respiratory medicine 2018-07, Vol.6 (7), p.545-553
Hauptverfasser: Rosenfeld, Margaret, Wainwright, Claire E, Higgins, Mark, Wang, Linda T, McKee, Charlotte, Campbell, Daniel, Tian, Simon, Schneider, Jennifer, Cunningham, Steve, Davies, Jane C, Wainwright, Claire E., Wang, Linda T., Davies, Jane C., Harris, William, Mogayzel, Peter, McCoy, Karen, Milla, Carlos, Rubenstein, Ronald, Walker, Seth, Black, Philip, Montgomery, Gregory, McColley, Susanna, Hiatt, Peter, Sawicki, Gregory, Rock, Michael, Aurora, Paul, Ratjen, Felix, Maitra, Anirban, Ives, Andrew, Gaillard, Erol, McNalley, Paul, Selvadurai, Hiranjan, Robinson, Philip
Format: Artikel
Sprache:eng
Schlagworte:
Aminophenols - therapeutic use
Australia
Canada
Chloride Channel Agonists - therapeutic use
Cystic Fibrosis - drug therapy
Cystic Fibrosis - genetics
Cystic Fibrosis Transmembrane Conductance Regulator - genetics
Female
Humans
Infant
Male
Mutation - genetics
Quinolones - therapeutic use
Treatment Outcome
United Kingdom
United States
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Beschreibung
Zusammenfassung:Ivacaftor is generally safe and effective in patients aged 2 years and older who have cystic fibrosis and specific CFTR mutations. We assessed its use in children aged 12 to
ISSN:2213-2600
2213-2619
DOI:10.1016/S2213-2600(18)30202-9