Results 231 to 240 of about 1,231,622 (330)

RFFL antisense oligonucleotides: A novel stabilization strategy to broaden the reach of CFTR modulator therapy. [PDF]

Mol Ther Nucleic Acids
Pedemonte N.
europepmc   +1 more source

Real-life impact of highly effective CFTR modulator therapy in children with cystic fibrosis. [PDF]

Front Pharmacol, 2023
Olivier M, Kavvalou A, Welsner M, Hirtz R, Straßburg S, Sutharsan S, Stehling F, Steindor M.   +7 more
europepmc   +1 more source

Transcriptomic and functional responses of the cystic fibrosis airway epithelium to CFTR modulator therapy. [PDF]

JCI Insight
Vladar EK, Gillen AE, Yadav S, Murphree MR, Baraghoshi D, Harris JK, Pruesse E, Niemiec SS, Wilson AW, Hisert KB, Humphries SM, Strand M, Lynch DA, Seibold MA, Beswick DM, Taylor-Cousar JL.   +15 more
europepmc   +1 more source

Functional evaluation of the CFTR activity of the Dele2-, Dele2,3- and Dele13-CFTR mutants on a heterologous expression system after treatment with pharmacological modulators.

Cristina Pastorino (20277087), Ludovica Menta (22258367), Emanuela Pesce (207804), Mariateresa Lena (20277090), Valeria Tomati (6080210), Valeria Capurro (282901), Marco Di Duca (11801237), Vito Terlizzi (5564168), Andrea Gramegna (5890742), Francesco Blasi (61418), Carlo Castellani (724942), Serena Cappato (115162), Federico Zara (176248), Nicoletta Pedemonte (383764), Renata Bocciardi (115174)   +14 more
openalex   +1 more source

Modulators of CFTR. Updates on clinical development and future directions

, 2021
Emmanuelle Bardin, Alexandra Pastor, Michaëla Semeraro, Anita Golec, Kate Hayes, B. Chevalier, Farouk Berhal, Guillaume Prestat, Alexandre Hinzpeter, Christine Gravier‐Pelletier, Iwona Pranke, Isabelle Sermet‐Gaudelus   +11 more
openalex   +2 more sources

Changes in Sleep in Children and Adults with Cystic Fibrosis and Primary Ciliary Dyskinesia over Time and after CFTR Modulator Therapy. [PDF]

J Clin Med, 2023
Cohen-Cymberknoh M, Lehavi M, Gileles-Hillel A, Atia O, Breuer O, Reiter J.   +5 more
europepmc   +1 more source

CFTR Modulators Therapy Efficacy in Reducing Cystic Fibrosis (CF) Exacerbation and Improving Selected Spirometry Parameters: A Real-Life Study in a Single-Centre Polish Population [PDF]

Hanna Maria Winiarska, Daria Springer, Filip Wojtaś, Ewa Wysocka, Szczepan Cofta   +4 more
openalex   +1 more source

CFTR modulator therapy for cystic fibrosis caused by the rare c.3700A>G mutation

, 2020
Puay‐Wah Phuan, Peter M. Haggie, Joseph Tan, Amber A. Rivera, Walter E. Finkbeiner, Dennis W. Nielson, Merlin Thomas, Ibrahim A. Janahi, A. S. Verkman   +8 more
openalex   +2 more sources

Theratyping as a tool to guide clinical decision-making in patients with suspected cystic fibrosis and variants currently ineligible for CFTR modulator therapy. [PDF]

Respir Med
Gannon RM, Wright BA, Bettis BA, Teresi ME, Fischer AJ, Weiner RL, Peña T, Hornick DB, Thornell IM, Rehman T, Pezzulo AA.   +10 more
europepmc   +1 more source

WS12.2 Evolution of inflammatory parameters during CFTR-modulator therapy with lumacaftor/ivacaftor in children aged 6 to 12 years

, 2021
Jochen Rößler, Rebecca L. Knoll, S. Wirsching, Maximiliano L. Cacicedo, Peter Mildenberger, Sonja Gehring, Oliver Nitsche, Krystyna Poplawska   +7 more
openalex   +1 more source