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Page 1
Cystic Fibrosis and Its Management Through Established and Emerging Therapies.
Spielberg DR, Clancy JP. Spielberg DR, et al. Among authors: clancy jp. Annu Rev Genomics Hum Genet. 2016 Aug 31;17:155-75. doi: 10.1146/annurev-genom-090314-050024. Epub 2016 Feb 22. Annu Rev Genomics Hum Genet. 2016. PMID: 26905785 Review.
CF treatments continue to advance with greater understanding of factors influencing long-term morbidity and mortality. In recent years, in-depth understanding of genetic and protein structure-function relationships has led to the introduction of targeted therapies for pati …
CF treatments continue to advance with greater understanding of factors influencing long-term morbidity and mortality. In recent year …
CFTR modulator theratyping: Current status, gaps and future directions.
Clancy JP, Cotton CU, Donaldson SH, Solomon GM, VanDevanter DR, Boyle MP, Gentzsch M, Nick JA, Illek B, Wallenburg JC, Sorscher EJ, Amaral MD, Beekman JM, Naren AP, Bridges RJ, Thomas PJ, Cutting G, Rowe S, Durmowicz AG, Mense M, Boeck KD, Skach W, Penland C, Joseloff E, Bihler H, Mahoney J, Borowitz D, Tuggle KL. Clancy JP, et al. J Cyst Fibros. 2019 Jan;18(1):22-34. doi: 10.1016/j.jcf.2018.05.004. Epub 2018 Jun 20. J Cyst Fibros. 2019. PMID: 29934203 Free PMC article. Review.
The theme of the workshop was centered on CFTR theratyping, a term that encompasses the use of CFTR modulators to define defects in CFTR in vitro, with application to both common and rare CFTR variants. ...
The theme of the workshop was centered on CFTR theratyping, a term that encompasses the use of CFTR modulators to define defects in C …
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. Among authors: clancy jp. 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 …
Pharmaceuticals targeting nonsense mutations in genetic diseases: progress in development.
Rowe SM, Clancy JP. Rowe SM, et al. Among authors: clancy jp. BioDrugs. 2009;23(3):165-74. doi: 10.2165/00063030-200923030-00003. BioDrugs. 2009. PMID: 19627168 Review.
Several of the published studies in cystic fibrosis have reported improvements in cystic fibrosis transmembrane conductance regulator (CFTR) biomarkers during short-term evaluation, including topical and systemic aminoglycoside treatment, and oral dosing with PTC124. ...
Several of the published studies in cystic fibrosis have reported improvements in cystic fibrosis transmembrane conductance regulator (CFTR) …
Long-term amikacin liposome inhalation suspension in cystic fibrosis patients with chronic P. aeruginosa infection.
Bilton D, Fajac I, Pressler T, Clancy JP, Sands D, Minic P, Cipolli M, Galeva I, Solé A, Quittner AL, Jumadilova Z, Ciesielska M, Konstan MW; CLEAR-110 Study Group. Bilton D, et al. Among authors: clancy jp. J Cyst Fibros. 2021 Nov;20(6):1010-1017. doi: 10.1016/j.jcf.2021.05.013. Epub 2021 Jun 16. J Cyst Fibros. 2021. PMID: 34144923 Free PMC article. Clinical Trial.
The CLEAR-110 extension study (ClinicalTrials.gov: NCT01316276; EudraCT: 2011-000443-24) assessed long-term safety, tolerability, and efficacy of ALIS in eligible patients who completed CLEAR-108. ...Mean change from baseline in sputum density of P. aeruginosa at day 672 w …
The CLEAR-110 extension study (ClinicalTrials.gov: NCT01316276; EudraCT: 2011-000443-24) assessed long-term safety, tolerability, and …
Long term effects of denufosol tetrasodium in patients with cystic fibrosis.
Ratjen F, Durham T, Navratil T, Schaberg A, Accurso FJ, Wainwright C, Barnes M, Moss RB; TIGER-2 Study Investigator Group. Ratjen F, et al. J Cyst Fibros. 2012 Dec;11(6):539-49. doi: 10.1016/j.jcf.2012.05.003. Epub 2012 Jun 8. J Cyst Fibros. 2012. PMID: 22682898 Free article. Clinical Trial.
Evaluating the Impact of Stopping Chronic Therapies after Modulator Drug Therapy in Cystic Fibrosis: The SIMPLIFY Clinical Trial Study Design.
Mayer-Hamblett N, Nichols DP, Odem-Davis K, Riekert KA, Sawicki GS, Donaldson SH, Ratjen F, Konstan MW, Simon N, Rosenbluth DB, Retsch-Bogart G, Clancy JP, VanDalfsen JM, Buckingham R, Gifford AH. Mayer-Hamblett N, et al. Among authors: clancy jp. Ann Am Thorac Soc. 2021 Aug;18(8):1397-1405. doi: 10.1513/AnnalsATS.202010-1336SD. Ann Am Thorac Soc. 2021. PMID: 33465316 Free PMC article.
The protocol is composed of two concurrent randomized controlled trials designed to evaluate the independent short-term effects of discontinuing hypertonic saline or dornase alfa, enabling individuals on both therapies to participate in one or both trials. ...
The protocol is composed of two concurrent randomized controlled trials designed to evaluate the independent short-term effects of di …
Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe disease.
Kishnani PS, Corzo D, Leslie ND, Gruskin D, Van der Ploeg A, Clancy JP, Parini R, Morin G, Beck M, Bauer MS, Jokic M, Tsai CE, Tsai BW, Morgan C, O'Meara T, Richards S, Tsao EC, Mandel H. Kishnani PS, et al. Among authors: clancy jp. Pediatr Res. 2009 Sep;66(3):329-35. doi: 10.1203/PDR.0b013e3181b24e94. Pediatr Res. 2009. PMID: 19542901 Free PMC article. Clinical Trial.
No significant differences in either safety or efficacy parameters were observed between the 20 and 40 mg/kg biweekly doses. Overall, long-term alglucosidase alpha treatment markedly extended survival as well as ventilation-free survival and improved cardiomyopathy....
No significant differences in either safety or efficacy parameters were observed between the 20 and 40 mg/kg biweekly doses. Overall, long- …
Safety and efficacy of alternative alglucosidase alfa regimens in Pompe disease.
Case LE, Bjartmar C, Morgan C, Casey R, Charrow J, Clancy JP, Dasouki M, DeArmey S, Nedd K, Nevins M, Peters H, Phillips D, Spigelman Z, Tifft C, Kishnani PS. Case LE, et al. Among authors: clancy jp. Neuromuscul Disord. 2015 Apr;25(4):321-32. doi: 10.1016/j.nmd.2014.12.004. Epub 2014 Dec 19. Neuromuscul Disord. 2015. PMID: 25617983 Free article. Clinical Trial.
Emerging phenotypes in long-term survivors with Pompe disease on standard enzyme replacement therapy (ERT) (alglucosidase alfa 20 mg/kg/2 weeks) can include patients with worsening motor function. ...
Emerging phenotypes in long-term survivors with Pompe disease on standard enzyme replacement therapy (ERT) (alglucosidase alfa 20 mg/ …
CFTR Modulator Therapies in Pediatric Cystic Fibrosis: Focus on Ivacaftor.
Kramer EL, Clancy JP. Kramer EL, et al. Among authors: clancy jp. Expert Opin Orphan Drugs. 2016 Oct;4(10):1033-1042. doi: 10.1080/21678707.2016.1226800. Epub 2016 Sep 13. Expert Opin Orphan Drugs. 2016. PMID: 28042521 Free PMC article.
Extending these therapies to young CF patients is proposed to have the greatest long term impact, potentially preventing later disease. AREAS COVERED: Here we summarize the research experience of CFTR modulators in pediatrics, focusing on ivacaftor and highlighting challen …
Extending these therapies to young CF patients is proposed to have the greatest long term impact, potentially preventing later diseas …
13 results