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Home > Therapeutic benefit observed with the CFTR potentiator, ivacaftor, in a CF patient homozygous for the W1282X CFTR nonsense mutation

Therapeutic benefit observed with the CFTR potentiator, ivacaftor, in a CF patient homozygous for the W1282X CFTR nonsense mutation

Resource Type

CF Research News

Authors

Venkateshwar Mutyam
Emily Falk Libby
Ning Peng
Denis Hadjiliadis
Michael Bonk
George M. Solomon
Steven M. Rowe

References

J Cyst Fibros. 2016 Oct 1. pii: S1569-1993(16)30607-5

Document

PDF icon CF Research News article [1]

Link

Scientific abstract [2]

Keywords

CFTR potentiator [3]
Ivacaftor [4]
PTC mutations [5]
Readthrough [6]
W1282X [7]

Date

Saturday, October 1, 2016

Featured resource article

Yes
Source URL:https://www.ecfs.eu/research-article/therapeutic-benefit-observed-cftr-potentiator-ivacaftor-cf-patient-homozygous

Links
[1] https://www.ecfs.eu/sites/default/files/cf-research-article-files/JCF-D-16-00207R1_FINAL_Rowe_15102016.pdf [2] https://www.ncbi.nlm.nih.gov/pubmed/?term=Therapeutic%20benefit%20observed%20with%20the%20CFTR%20potentiator%2C%20ivacaftor%2C%20in%20a%20CF%20patient%20homozygous%20for%20the%20W1282X%20CFTR%20nonsense%20mutation [3] https://www.ecfs.eu/research-article-keywords/cftr-potentiator [4] https://www.ecfs.eu/research-article-keywords/ivacaftor [5] https://www.ecfs.eu/research-article-keywords/ptc-mutations [6] https://www.ecfs.eu/research-article-keywords/readthrough [7] https://www.ecfs.eu/research-article-keywords/w1282x