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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 Mutation: A Phase 3b, Randomized, Placebo-controlled Study.
Mall MA, Brugha R, Gartner S, Legg J, Moeller A, Mondejar-Lopez P, Prais D, Pressler T, Ratjen F, Reix P, Robinson PD, Selvadurai H, Stehling F, Ahluwalia N, Arteaga-Solis E, Bruinsma BG, Jennings M, Moskowitz SM, Noel S, Tian S, Weinstock TG, Wu P, Wainwright CE, Davies JC. Mall MA, et al. Among authors: prais d. Am J Respir Crit Care Med. 2022 Dec 1;206(11):1361-1369. doi: 10.1164/rccm.202202-0392OC. Am J Respir Crit Care Med. 2022. PMID: 35816621 Free PMC article. Clinical Trial.
VX-659-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles.
Davies JC, Moskowitz SM, Brown C, Horsley A, Mall MA, McKone EF, Plant BJ, Prais D, Ramsey BW, Taylor-Cousar JL, Tullis E, Uluer A, McKee CM, Robertson S, Shilling RA, Simard C, Van Goor F, Waltz D, Xuan F, Young T, Rowe SM; VX16-659-101 Study Group. Davies JC, et al. Among authors: prais d. N Engl J Med. 2018 Oct 25;379(17):1599-1611. doi: 10.1056/NEJMoa1807119. Epub 2018 Oct 18. N Engl J Med. 2018. PMID: 30334693 Free PMC article. Clinical Trial.
Long-term safety and efficacy of tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years or older who are homozygous or heterozygous for Phe508del CFTR (EXTEND): an open-label extension study.
Flume PA, Biner RF, Downey DG, Brown C, Jain M, Fischer R, De Boeck K, Sawicki GS, Chang P, Paz-Diaz H, Rubin JL, Yang Y, Hu X, Pasta DJ, Millar SJ, Campbell D, Wang X, Ahluwalia N, Owen CA, Wainwright CE; VX14-661-110 study group. Flume PA, et al. Lancet Respir Med. 2021 Jul;9(7):733-746. doi: 10.1016/S2213-2600(20)30510-5. Epub 2021 Feb 10. Lancet Respir Med. 2021. PMID: 33581080
Evaluation of sputum cultures in children with spinal Muscular atrophy.
Levine H, Nevo Y, Katz J, Mussaffi H, Chodick G, Mei-Zahav M, Stafler P, Steuer G, Bar-On O, Mantin H, Prais D, Aharoni S. Levine H, et al. Among authors: prais d. Respir Med. 2023 Apr;209:107143. doi: 10.1016/j.rmed.2023.107143. Epub 2023 Feb 8. Respir Med. 2023. PMID: 36764497
Eradication of Nontuberculous Mycobacteria in People with Cystic Fibrosis Treated with Elexacaftor/Tezacaftor/Ivacaftor: A Multicenter Cohort Study.
Wiesel V, Aviram M, Mei-Zahav M, Dotan M, Prais D, Cohen-Cymberknoh M, Gur M, Bar-Yoseph R, Livnat G, Goldbart A, Hazan G, Hazan I, Golan-Tripto I. Wiesel V, et al. Among authors: prais d. J Cyst Fibros. 2024 Jan;23(1):41-49. doi: 10.1016/j.jcf.2023.05.003. Epub 2023 May 10. J Cyst Fibros. 2024. PMID: 37173154
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