The future of cystic fibrosis care: a global perspective

SC Bell, MA Mall, H Gutierrez, M Macek… - The Lancet …, 2020 - thelancet.com
Executive summary The past six decades have seen remarkable improvements in health
outcomes for people with cystic fibrosis, which was once a fatal disease of infants and young …

[HTML][HTML] CFTR modulators: the changing face of cystic fibrosis in the era of precision medicine

M Lopes-Pacheco - Frontiers in pharmacology, 2020 - frontiersin.org
Cystic fibrosis (CF) is a lethal inherited disease caused by mutations in the CF
transmembrane conductance regulator (CFTR) gene, which result in impairment of CFTR …

Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double …

HGM Heijerman, EF McKone, DG Downey… - The Lancet, 2019 - thelancet.com
Background Cystic fibrosis transmembrane conductance regulator (CFTR) modulators
correct the basic defect caused by CFTR mutations. Improvements in health outcomes have …

[HTML][HTML] Future therapies for cystic fibrosis

L Allen, L Allen, SB Carr, G Davies, D Downey… - Nature …, 2023 - nature.com
We are currently witnessing transformative change for people with cystic fibrosis with the
introduction of small molecule, mutation-specific drugs capable of restoring function of the …

Rapid improvement after starting elexacaftor–tezacaftor–ivacaftor in patients with cystic fibrosis and advanced pulmonary disease

PR Burgel, I Durieu, R Chiron, S Ramel… - American journal of …, 2021 - atsjournals.org
Rationale: Elexacaftor–tezacaftor–ivacaftor is a CFTR (cystic fibrosis [CF] transmembrane
conductance regulator) modulator combination, developed for patients with CF with at least …

Cystic fibrosis in the year 2020: A disease with a new face

K De Boeck - Acta paediatrica, 2020 - Wiley Online Library
The autosomal recessive disease cystic fibrosis (CF) was once untreatable and deadly in
childhood, but now most patients survive to adulthood. Many countries have instituted CF …

Cystic fibrosis: a review

T Ong, BW Ramsey - Jama, 2023 - jamanetwork.com
Importance Cystic fibrosis, a genetic disorder defined by variants in the cystic fibrosis
transmembrane conductance regulator (CFTR) gene, affects more than 30 000 individuals in …

On-Line Analysis of Exhaled Breath: Focus Review

T Bruderer, T Gaisl, MT Gaugg, N Nowak… - Chemical …, 2019 - ACS Publications
On-line analysis of exhaled breath offers insight into a person's metabolism without the need
for sample preparation or sample collection. Due to its noninvasive nature and the possibility …

21st century mathematics learning challenges: Bibliometric analysis of trends and best practices in shinta indexed scientific publications

ND Safitri, R Darmayanti, U Usmiyatun… - JEMS: Jurnal …, 2023 - e-journal.unipma.ac.id
This study aims to find out the issues or problems that become the direction and trends of
mathematics education research as an effort made by teachers in responding to challenges …

Nonantimicrobial actions of macrolides: overview and perspectives for future development

JA Kricker, CP Page, FR Gardarsson… - Pharmacological …, 2021 - ASPET
Macrolides are among the most widely prescribed broad spectrum antibacterials, particularly
for respiratory infections. It is now recognized that these drugs, in particular azithromycin …