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Year Number of Results
2001 1
2010 1
2011 1
2013 2
2014 1
2016 1
2017 1
2018 1
2020 1
2021 4
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2023 4
2024 0

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18 results

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Page 1
Long-Term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged 6 Years with Cystic Fibrosis and at Least One F508del Allele: A Phase 3, Open-Label Clinical Trial.
Wainwright C, McColley SA, McNally P, Powers M, Ratjen F, Rayment JH, Retsch-Bogart G, Roesch E, Ahluwalia N, Chin A, Chu C, Lu M, Menon P, Waltz D, Weinstock T, Zelazoski L, Davies JC. Wainwright C, et al. Among authors: davies jc. Am J Respir Crit Care Med. 2023 Jul 1;208(1):68-78. doi: 10.1164/rccm.202301-0021OC. Am J Respir Crit Care Med. 2023. PMID: 37154609
Rationale: A 24-week, phase 3, open-label study showed elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was safe and efficacious in children aged 6-11 years with cystic fibrosis (CF) and one or more F508del-CFTR alleles. Objectives: To assess long-term safety and efficacy of …
Rationale: A 24-week, phase 3, open-label study showed elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was safe and efficacious in children a …
Progress in Respiratory Gene Therapy.
McLachlan G, Alton EWFW, Boyd AC, Clarke NK, Davies JC, Gill DR, Griesenbach U, Hickmott JW, Hyde SC, Miah KM, Molina CJ. McLachlan G, et al. Among authors: davies jc. Hum Gene Ther. 2022 Sep;33(17-18):893-912. doi: 10.1089/hum.2022.172. Hum Gene Ther. 2022. PMID: 36074947 Free PMC article. Review.
In addition, the inclusion of features such as codon optimization and promoters providing long-term expression have improved the expression characteristics of therapeutic transgenes. ...
In addition, the inclusion of features such as codon optimization and promoters providing long-term expression have improved the expr …
Entering the era of highly effective modulator therapies.
Dave K, Dobra R, Scott S, Saunders C, Matthews J, Simmonds NJ, Davies JC. Dave K, et al. Among authors: davies jc. Pediatr Pulmonol. 2021 Feb;56 Suppl 1:S79-S89. doi: 10.1002/ppul.24968. Pediatr Pulmonol. 2021. PMID: 33434412 Review.
It is highly effective with impressive clinical impact in the lungs and gastrointestinal tract; longer-term data from patient registries show fewer exacerbations, a slower rate of lung function loss and reduced need for transplantation in patients receiving ivacaftor. ...T …
It is highly effective with impressive clinical impact in the lungs and gastrointestinal tract; longer-term data from patient registr …
Restoration of exocrine pancreatic function in older children with cystic fibrosis on ivacaftor.
Nichols AL, Davies JC, Jones D, Carr SB. Nichols AL, et al. Among authors: davies jc. Paediatr Respir Rev. 2020 Sep;35:99-102. doi: 10.1016/j.prrv.2020.04.003. Epub 2020 Apr 14. Paediatr Respir Rev. 2020. PMID: 32386958 Review.
Although the effect of ivacaftor on pancreatic function may be more significant in younger children, evidence is mounting that there may still be potential for improvement in older children on long term therapy....
Although the effect of ivacaftor on pancreatic function may be more significant in younger children, evidence is mounting that there may sti …
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, extension study.
Chilvers MA, Davies JC, Milla C, Tian S, Han Z, Cornell AG, Owen CA, Ratjen F. Chilvers MA, et al. Among authors: davies jc. Lancet Respir Med. 2021 Jul;9(7):721-732. doi: 10.1016/S2213-2600(20)30517-8. Epub 2021 Jan 28. Lancet Respir Med. 2021. PMID: 33516285 Clinical Trial.
BACKGROUND: The safety and efficacy of 24 weeks of lumacaftor-ivacaftor combination therapy in children aged 6-11 years with cystic fibrosis homozygous for the F508del-CFTR mutation was previously shown in two phase 3 studies. Here, we report long-term safety and efficacy …
BACKGROUND: The safety and efficacy of 24 weeks of lumacaftor-ivacaftor combination therapy in children aged 6-11 years with cystic fibrosis …
A Short extension to multiple breath washout provides additional signal of distal airway disease in people with CF: A pilot study.
Short C, Semple T, Saunders C, Hughes D, Irving S, Gardener L, Rosenthal M, Robinson PD, Davies JC. Short C, et al. Among authors: davies jc. J Cyst Fibros. 2022 Jan;21(1):146-154. doi: 10.1016/j.jcf.2021.06.013. Epub 2021 Jul 16. J Cyst Fibros. 2022. PMID: 34275757 Free article.
METHODS: We conducted a pilot study on people undergoing MBW tests: 10 healthy controls (HC) and 43 cystic fibrosis (CF) subjects performed an SVC after the standard end of test. We term the new outcome LCI with Short extension (LCI(ShX)). We assessed (i) CF/ HC difference …
METHODS: We conducted a pilot study on people undergoing MBW tests: 10 healthy controls (HC) and 43 cystic fibrosis (CF) subjects performed …
A Phase 3, open-label, 96-week trial to study the safety, tolerability, and efficacy of tezacaftor/ivacaftor in children ≥ 6 years of age homozygous for F508del or heterozygous for F508del and a residual function CFTR variant.
Sawicki GS, Chilvers M, McNamara J, Naehrlich L, Saunders C, Sermet-Gaudelus I, Wainwright CE, Ahluwalia N, Campbell D, Harris RS, Paz-Diaz H, Shih JL, Davies JC. Sawicki GS, et al. Among authors: davies jc. J Cyst Fibros. 2022 Jul;21(4):675-683. doi: 10.1016/j.jcf.2022.02.003. Epub 2022 Feb 18. J Cyst Fibros. 2022. PMID: 35190292 Free article. Clinical Trial.
CONCLUSIONS: Tezacaftor/ivacaftor is generally safe and well tolerated, and treatment effects are maintained for up to 120 weeks. These results support long-term use of tezacaftor/ivacaftor in children 6 years of age with CF and F/F or F/RF genotypes....
CONCLUSIONS: Tezacaftor/ivacaftor is generally safe and well tolerated, and treatment effects are maintained for up to 120 weeks. These resu …
Computed cardiopulmonography and the idealized lung clearance index, iLCI2.5, in early-stage cystic fibrosis.
Sandhu D, Redmond JL, Smith NMJ, Short C, Saunders CJ, Couper JH, Fullerton CJ, Richmond G, Talbot NP, Davies JC, Ritchie GAD, Robbins PA. Sandhu D, et al. Among authors: davies jc. J Appl Physiol (1985). 2023 Jul 1;135(1):205-216. doi: 10.1152/japplphysiol.00744.2022. Epub 2023 Jun 1. J Appl Physiol (1985). 2023. PMID: 37262105 Free PMC article.
Both a sensitivity analysis on iLCI(2.5) and a regression analysis on LCI(2.5) revealed that these depended primarily on an interactive term between CCP parameters of the form sigmalnCL*(DS/FRC). In conclusion, the LCI(2.5) (or iLCI(2.5)) probably reflects an amalgam of di …
Both a sensitivity analysis on iLCI(2.5) and a regression analysis on LCI(2.5) revealed that these depended primarily on an interactive t …
Recovery of lung function following a pulmonary exacerbation in patients with cystic fibrosis and the G551D-CFTR mutation treated with ivacaftor.
Flume PA, Wainwright CE, Elizabeth Tullis D, Rodriguez S, Niknian M, Higgins M, Davies JC, Wagener JS. Flume PA, et al. Among authors: davies jc. J Cyst Fibros. 2018 Jan;17(1):83-88. doi: 10.1016/j.jcf.2017.06.002. Epub 2017 Jun 24. J Cyst Fibros. 2018. PMID: 28651844 Free article.
Short-term recovery was measured 2 to 8weeks after treatment, and long-term recovery was determined at the end-of-study, both compared with baseline measured just prior to the PEx. RESULTS: Fewer patients receiving ivacaftor experienced a PEx than patients receiving …
Short-term recovery was measured 2 to 8weeks after treatment, and long-term recovery was determined at the end-of-study, both …
18 results