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

Journal of Cystic Fibrosis - Tập 16 - Trang 24-29 - 2017
Venkateshwar Mutyam1, Emily Falk Libby2, Ning Peng1, Denis Hadjiliadis3, Michael Bonk3, George M. Solomon1,2, Steven M. Rowe1,4,5,2
1Department of Medicine, University of Alabama at Birmingham, Birmingham, AL, USA
2Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, AL, USA
3Department of Medicine, University of Pennsylvania Medical Center, PA, USA
4Department of Pediatrics, University of Alabama at Birmingham, Birmingham, AL USA
5Department of Cell Developmental and Integrative Biology, University of Alabama at Birmingham, Birmingham, AL, USA

Tài liệu tham khảo

Thông tin
Thông tin xuất bản

Journal of Cystic Fibrosis

Tập 16

24-29

Thông tin tác giả