| Elexacaftor–tezacaftor–ivacaftor for cystic fibrosis with a single Phe508del allele PG Middleton, MA Mall, P Dřevínek, LC Lands, EF McKone, D Polineni, ... New England Journal of Medicine 381 (19), 1809-1819, 2019 | 1776 | 2019 |
| Efficacy and safety of lumacaftor and ivacaftor in patients aged 6–11 years with cystic fibrosis homozygous for F508del-CFTR: a randomised, placebo-controlled phase 3 trial F Ratjen, C Hug, G Marigowda, S Tian, X Huang, S Stanojevic, CE Milla, ... The lancet Respiratory medicine 5 (7), 557-567, 2017 | 342 | 2017 |
| Assessment of safety and efficacy of long-term treatment with combination lumacaftor and ivacaftor therapy in patients with cystic fibrosis homozygous for the F508del-CFTR … MW Konstan, EF McKone, RB Moss, G Marigowda, S Tian, D Waltz, ... The lancet Respiratory medicine 5 (2), 107-118, 2017 | 331 | 2017 |
| 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 M Rosenfeld, CE Wainwright, M Higgins, LT Wang, C McKee, D Campbell, ... The lancet respiratory medicine 6 (7), 545-553, 2018 | 261 | 2018 |
| Disease progression in patients with cystic fibrosis treated with ivacaftor: data from national US and UK registries N Volkova, K Moy, J Evans, D Campbell, S Tian, C Simard, M Higgins, ... Journal of Cystic Fibrosis 19 (1), 68-79, 2020 | 253 | 2020 |
| Triple Therapy for Cystic Fibrosis Phe508del–Gating and –Residual Function Genotypes PJ Barry, MA Mall, A Álvarez, C Colombo, KM de Winter-De Groot, I Fajac, ... New England Journal of Medicine 385 (9), 815-825, 2021 | 218 | 2021 |
| A Phase 3 Open-Label Study of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 through 11 Years of Age with Cystic Fibrosis and at Least One F508del Allele ET Zemanick, JL Taylor-Cousar, J Davies, RL Gibson, MA Mall, ... American journal of respiratory and critical care medicine 203 (12), 1522-1532, 2021 | 217 | 2021 |
| Data from the US and UK cystic fibrosis registries support disease modification by CFTR modulation with ivacaftor L Bessonova, N Volkova, M Higgins, L Bengtsson, S Tian, C Simard, ... Thorax 73 (8), 731-740, 2018 | 215 | 2018 |
| Safety, pharmacokinetics, and pharmacodynamics of lumacaftor and ivacaftor combination therapy in children aged 2–5 years with cystic fibrosis homozygous for F508del-CFTR: an … JJ McNamara, SA McColley, G Marigowda, F Liu, S Tian, CA Owen, ... The Lancet Respiratory Medicine 7 (4), 325-335, 2019 | 139 | 2019 |
| An open-label extension study of ivacaftor in children with CF and a CFTR gating mutation initiating treatment at age 2–5 years (KLIMB) M Rosenfeld, S Cunningham, WT Harris, A Lapey, WE Regelmann, ... Journal of Cystic Fibrosis 18 (6), 838-843, 2019 | 118 | 2019 |
| Efficacy and safety of elexacaftor plus tezacaftor plus ivacaftor versus tezacaftor plus ivacaftor in people with cystic fibrosis homozygous for F508del-CFTR: a 24-week … S Sutharsan, EF McKone, DG Downey, J Duckers, G MacGregor, E Tullis, ... The Lancet Respiratory Medicine 10 (3), 267-277, 2022 | 100 | 2022 |
| Ivacaftor in infants aged 4 to< 12 months with cystic fibrosis and a gating mutation. Results of a two-part phase 3 clinical trial JC Davies, CE Wainwright, GS Sawicki, MN Higgins, D Campbell, ... American journal of respiratory and critical care medicine 203 (5), 585-593, 2021 | 87 | 2021 |
| Lumacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease homozygous for F508del-CFTR JL Taylor-Cousar, M Jain, TL Barto, T Haddad, J Atkinson, S Tian, R Tang, ... Journal of Cystic Fibrosis 17 (2), 228-235, 2018 | 83 | 2018 |
| Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 Through 11 Years of Age with Cystic Fibrosis Heterozygous for F508del and a Minimal Function … MA Mall, R Brugha, S Gartner, J Legg, A Moeller, P Mondejar-Lopez, ... American journal of respiratory and critical care medicine 206 (11), 1361-1369, 2022 | 81 | 2022 |
| Long-term safety of lumacaftor–ivacaftor in children aged 2–5 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a multicentre, phase 3, open-label, extension … JE Hoppe, M Chilvers, F Ratjen, JJ McNamara, CA Owen, S Tian, ... The Lancet Respiratory Medicine 9 (9), 977-988, 2021 | 42 | 2021 |
| Long-term safety and efficacy of lumacaftor–ivacaftor therapy in children aged 6–11 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a phase 3, open-label … MA Chilvers, JC Davies, C Milla, S Tian, Z Han, AG Cornell, CA Owen, ... The Lancet Respiratory Medicine 9 (7), 721-732, 2021 | 36 | 2021 |
| Long-term safety and efficacy of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis and at least one F508del allele: 144-week interim results from a 192-week open … CL Daines, E Tullis, S Costa, RW Linnemann, MA Mall, EF McKone, ... European Respiratory Journal 62 (6), 2023 | 30 | 2023 |
| Safety and efficacy of vanzacaftor–tezacaftor–deutivacaftor in adults with cystic fibrosis: randomised, double-blind, controlled, phase 2 trials AZ Uluer, G MacGregor, P Azevedo, V Indihar, C Keating, MA Mall, ... The Lancet Respiratory Medicine 11 (6), 550-562, 2023 | 28 | 2023 |
| VO2max as an exercise tolerance endpoint in people with cystic fibrosis: Lessons from a lumacaftor/ivacaftor trial J Wilson, X You, M Ellis, DS Urquhart, L Jha, M Duncan, S Tian, RA Harris, ... Journal of Cystic Fibrosis 20 (3), 499-505, 2021 | 26 | 2021 |
| Elexacaftor/tezacaftor/ivacaftor treatment and depression-related events B Ramsey, CU Correll, DR DeMaso, E McKone, E Tullis, ... American Journal of Respiratory and Critical Care Medicine 209 (3), 299-306, 2024 | 25 | 2024 |
