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2011 1
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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-label extension study.
Daines CL, Tullis E, Costa S, Linnemann RW, Mall MA, McKone EF, Polineni D, Quon BS, Ringshausen FC, Rowe SM, Selvadurai H, Taylor-Cousar JL, Withers NJ, Ahluwalia N, Moskowitz SM, Prieto-Centurion V, Tan YV, Tian S, Weinstock T, Xuan F, Zhang Y, Ramsey B, Griese M; VX17-445-105 Study Group. Daines CL, et al. Eur Respir J. 2023 Dec 7;62(6):2202029. doi: 10.1183/13993003.02029-2022. Print 2023 Dec. Eur Respir J. 2023. PMID: 37945033 Free PMC article. Clinical Trial.
BACKGROUND: In two pivotal phase 3 trials, up to 24 weeks of treatment with elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was efficacious and safe in patients with cystic fibrosis (CF) 12 years of age who have at least one F508del allele. The aim of this study is to assess long- …
BACKGROUND: In two pivotal phase 3 trials, up to 24 weeks of treatment with elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was efficacious a …
Use of elexacaftor/tezacaftor/ivacaftor among cystic fibrosis lung transplant recipients.
Ramos KJ, Guimbellot JS, Valapour M, Bartlett LE, Wai TH, Goss CH, Pilewski JM, Faro A, Diamond JM; CFLTC Study Group. Ramos KJ, et al. J Cyst Fibros. 2022 Sep;21(5):745-752. doi: 10.1016/j.jcf.2022.04.009. Epub 2022 Apr 23. J Cyst Fibros. 2022. PMID: 35474016 Free PMC article.
Further study is needed to determine the risks and benefits of ETI in the CF lung transplant population given the potential for drug interactions, side effects leading to discontinuation of ETI, and the possible mechanisms for ETI to positively impact long-term post-transp …
Further study is needed to determine the risks and benefits of ETI in the CF lung transplant population given the potential for drug interac …
Effects of a Partially Supervised Conditioning Program in Cystic Fibrosis: An International Multicenter, Randomized Controlled Trial (ACTIVATE-CF).
Hebestreit H, Kriemler S, Schindler C, Stein L, Karila C, Urquhart DS, Orenstein DM, Lands LC, Schaeff J, Eber E, Radtke T; ACTIVATE-CF Study Working Group. Hebestreit H, et al. Am J Respir Crit Care Med. 2022 Feb 1;205(3):330-339. doi: 10.1164/rccm.202106-1419OC. Am J Respir Crit Care Med. 2022. PMID: 34735776 Free PMC article. Clinical Trial.
Rationale: The long-term effects of vigorous physical activity (PA) on lung function in cystic fibrosis are unclear. ...
Rationale: The long-term effects of vigorous physical activity (PA) on lung function in cystic fibrosis are unclear. ...
Transcriptional firing represses bactericidal activity in cystic fibrosis airway neutrophils.
Margaroli C, Moncada-Giraldo D, Gulick DA, Dobosh B, Giacalone VD, Forrest OA, Sun F, Gu C, Gaggar A, Kissick H, Wu R, Gibson G, Tirouvanziam R. Margaroli C, et al. Cell Rep Med. 2021 Apr 8;2(4):100239. doi: 10.1016/j.xcrm.2021.100239. eCollection 2021 Apr 20. Cell Rep Med. 2021. PMID: 33948572 Free PMC article.
Treatment by the RNA polymerase II and III inhibitor alpha-amanitin restores the expression of key antimicrobial genes and increases the bactericidal capacity of CF airway neutrophils in vitro and in short-term sputum cultures ex vivo. Broadly, our findings show that neutr …
Treatment by the RNA polymerase II and III inhibitor alpha-amanitin restores the expression of key antimicrobial genes and increases the bac …
PROMISE: Working with the CF community to understand emerging clinical and research needs for those treated with highly effective CFTR modulator therapy.
Nichols DP, Donaldson SH, Frederick CA, Freedman SD, Gelfond D, Hoffman LR, Kelly A, Narkewicz MR, Pittman JE, Ratjen F, Sagel SD, Rosenfeld M, Schwarzenberg SJ, Singh PK, Solomon GM, Stalvey MS, Kirby S, VanDalfsen JM, Clancy JP, Rowe SM. Nichols DP, et al. J Cyst Fibros. 2021 Mar;20(2):205-212. doi: 10.1016/j.jcf.2021.02.003. Epub 2021 Feb 19. J Cyst Fibros. 2021. PMID: 33619012 Free PMC article. Review.
Multiple observational research studies are now being conducted to better understand the impacts of this important therapeutic milestone on long-term outcomes, patient care needs, and future research priorities. ...
Multiple observational research studies are now being conducted to better understand the impacts of this important therapeutic milestone on …
Long term clinical effectiveness of ivacaftor in people with the G551D CFTR mutation.
Guimbellot JS, Baines A, Paynter A, Heltshe SL, VanDalfsen J, Jain M, Rowe SM, Sagel SD; GOAL-e2 Investigators. Guimbellot JS, et al. J Cyst Fibros. 2021 Mar;20(2):213-219. doi: 10.1016/j.jcf.2020.11.008. Epub 2020 Nov 25. J Cyst Fibros. 2021. PMID: 33249004 Free PMC article.
BACKGROUND: The cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, ivacaftor, was first approved for people with CF and the G551D CFTR mutation. This study describes the long-term clinical effectiveness of ivacaftor in this population. METHODS: We cond …
BACKGROUND: The cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, ivacaftor, was first approved for people with CF and …
Bronchopulmonary dysplasia is associated with reduced oral nitrate reductase activity in extremely preterm infants.
Gentle SJ, Ahmed KA, Yi N, Morrow CD, Ambalavanan N, Lal CV, Patel RP. Gentle SJ, et al. Redox Biol. 2021 Jan;38:101782. doi: 10.1016/j.redox.2020.101782. Epub 2020 Nov 3. Redox Biol. 2021. PMID: 33166868 Free PMC article.
We characterized NR activity in extremely preterm infants from birth until 34 weeks' post menstrual age (PMA), determined whether changes in the oral microbiome contribute to changes in NR activity, and determined whether changes in NR activity correlated with disease. ...
We characterized NR activity in extremely preterm infants from birth until 34 weeks' post menstrual age (PMA), determined whether cha …
Gaming Console Home-Based Exercise for Adults with Cystic Fibrosis: Study Protocol.
Lowman JD, Solomon GM, Rowe SM, Yuen HK. Lowman JD, et al. Int J Caring Sci. 2020 Spring/Summer;13(2):1530-1540. Int J Caring Sci. 2020. PMID: 33163109 Free PMC article.
METHODS: A single group pretest-posttest design will be used to examine the immediate (12-weeks) and long-term effect (24-weeks) of a home-based exergame program on improving pulmonary-related function (physical fitness and respiratory function) in sedentary adults with CF …
METHODS: A single group pretest-posttest design will be used to examine the immediate (12-weeks) and long-term effect (24-weeks) of a …
Airway nitrite is increased in extremely preterm infants with bronchopulmonary dysplasia.
Gentle SJ, Freeman A, Patel RP, Ambalavanan N, Lal CV. Gentle SJ, et al. Respir Res. 2020 Sep 21;21(1):244. doi: 10.1186/s12931-020-01508-8. Respir Res. 2020. PMID: 32957939 Free PMC article.
METHODS: This was a prospective cohort study of extremely low birth infants (< 28 weeks' gestation) at the University of Alabama at Birmingham. Nitrite levels from tracheal aspirates (TAs) were compared between intubated and ventilated infants with BPD and gestation mat …
METHODS: This was a prospective cohort study of extremely low birth infants (< 28 weeks' gestation) at the University of Alabama a …
Haemophilus influenzae persists in biofilm communities in a smoke-exposed ferret model of COPD.
Hunt BC, Stanford D, Xu X, Li J, Gaggar A, Rowe SM, Raju SV, Swords WE. Hunt BC, et al. ERJ Open Res. 2020 Aug 11;6(3):00200-2020. doi: 10.1183/23120541.00200-2020. eCollection 2020 Jul. ERJ Open Res. 2020. PMID: 32802827 Free PMC article.
METHODS: In this study, we used a recently described ferret model for COPD, in which animals undergo chronic long-term exposure to cigarette smoke, to define host-pathogen interactions during COPD-related NTHi infections. ...
METHODS: In this study, we used a recently described ferret model for COPD, in which animals undergo chronic long-term exposure to ci …
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