RFFL-mediated protein quality control limits functional rescue of TRID-CFTR modulator combination therapy for cystic fibrosis nonsense mutations. [PDF]
Tateishi H +3 more
europepmc +1 more source
The impact of CFTR modulator triple therapy on type 2 inflammatory response in patients with cystic fibrosis. [PDF]
Mehta AM +8 more
europepmc +1 more source
Quantitative Systems Toxicology Predicts Ivacaftor-Induced Oxidative Stress Contributes to CFTR Modulator Hepatotoxicity. [PDF]
Shi A, Cornwell C, Yang K, Beringer PM.
europepmc +1 more source
Short-term monitoring of CFTR modulator therapy in adults and children with cystic fibrosis using low and ultra-low-dose lung CT. [PDF]
Maccioni F +9 more
europepmc +1 more source
Real-life impact of highly effective CFTR modulator therapy in children with cystic fibrosis. [PDF]
Olivier M +7 more
europepmc +1 more source
Theratyping as a tool to guide clinical decision-making in patients with suspected cystic fibrosis and variants currently ineligible for CFTR modulator therapy. [PDF]
Gannon RM +10 more
europepmc +1 more source
Changes in Sleep in Children and Adults with Cystic Fibrosis and Primary Ciliary Dyskinesia over Time and after CFTR Modulator Therapy. [PDF]
Cohen-Cymberknoh M +5 more
europepmc +1 more source
Respiratory Muscle Function in Children and Adolescents with Cystic Fibrosis in the Era of CFTR Modulator Therapies. [PDF]
García-Pérez-de-Sevilla G +8 more
europepmc +1 more source
Antisense oligonucleotide targeting the E3 ligase RFFL potentiates CFTR modulator efficacy in CF primary bronchial epithelial cells. [PDF]
Hinata D +9 more
europepmc +1 more source
CFTR modulator therapy drives microbiome restructuring through improved host physiology in cystic fibrosis: the IMMProveCF phase IV trial. [PDF]
Knoll RL +16 more
europepmc +1 more source

