Results 141 to 150 of about 11,406 (228)

In utero and postnatal ivacaftor/lumacaftor therapy rescues multiorgan disease in CFTR-F508del ferrets

JCI Insight
Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator (CFTR) gene, with F508del being the most prevalent mutation.
Idil Apak Evans, Xingshen Sun, Bo Liang, Amber R. Vegter, Lydia Guo, Thomas J. Lynch, Yan Zhang, Yulong Zhang, Yaling Yi, Yu Yang, Zehua Feng, Soo Yeun Park, Amanita Shonka, Hannah McCumber, Lisi Qi, Peipei Wu, Guangming Liu, Allison Lacina, Kai Wang, Katherine N. Gibson-Corley, David K. Meyerholz, Dominique H. Limoli, Bradley H. Rosen, Ziying Yan, Douglas J. Bartels, John F. Engelhardt   +25 more
doaj   +1 more source

Profile of tezacaftor/ivacaftor combination and its potential in the treatment of cystic fibrosis

Therapeutics and Clinical Risk Management, 2019
Dejene Shiferaw,* Shoaib Faruqi*Department of Respiratory Medicine, Hull University Teaching Hospitals NHS Trust, Cottingham HU16 5JQ, UK *These authors contributed equally to this workCorrespondence: Shoaib FaruqiDepartment of Respiratory Medicine ...
Shiferaw D, Faruqi S
doaj  

The PROSPECT Is Bright for CFTR Modulators

Annals of the American Thoracic Society, 2021
Kristina Montemayor, Noah Lechtzin
openaire   +2 more sources

Beyond BMI: Nutritional Recovery and Functional Implications of CFTR Modulators in Cystic Fibrosis. [PDF]

Biology (Basel)
Linguiti G, Granberg V, Leonetti G, Lassandro G, Lassandro M, Delvecchio M, Giordano P.   +6 more
europepmc   +1 more source

CFTR modulators partially restore the epithelial interferome in Aspergillus infection to improve clinical outcome. [PDF]

EBioMedicine
Laverty SL, Felton I, Casey M, Hopwood B, Simmonds N, Armstrong-James D, Shah A, Kelleher P.   +7 more
europepmc   +1 more source

L467F;F508del Complex Allele in a Heterozygous State with CFTRdele2,3: What to Expect from CFTR Modulators? [PDF]

Int J Mol Sci
Kondratyeva E, Efremova A, Melyanovskaya Y, Krasnova M, Milovanov M, Shchagina O, Stepanova A, Voronkova A, Sherman V, Kutsev S, Goldshtein D.   +10 more
europepmc   +1 more source

Breaking boundaries or a small piece of the puzzle: can CFTR modulators transform bronchiectasis care? [PDF]

ERJ Open Res
Graeber SY, Frost F.
europepmc   +1 more source

Editorial: Real-world experience with CFTR modulator therapy

Frontiers in Pharmacology, 2023
Burkhard Tümmler, Burkhard Tümmler, Pierre-Régis Burgel, Pierre-Régis Burgel   +3 more
openaire   +3 more sources

Phosphatidylserine liposomes for <i>Mycobacterium abscessus</i> infections management in people with cystic fibrosis non-eligible for CFTR modulators. [PDF]

Front Immunol
Olimpieri T, Poerio N, Saliu F, Lorè NI, Ciciriello F, Ponsecchi G, Alghisi F, Cirillo DM, D'Andrea MM, Fraziano M.   +9 more
europepmc   +1 more source

WS13.2 Novel F508del-CFTR corrector that complements other CFTR modulators

Journal of Cystic Fibrosis, 2016
J.P. Miller, L. Drew, C.M. Bastos, D.J. Parks, O. Green, B. McEwan, N. Patel, S. Aghamohammadzadeh, A. Villella, K.A. Giuliano, K.A. Longo, M. Ivarsson, B. Munoz, H. Mehmet   +13 more
openaire   +1 more source