Review of CFTR modulators 2020.
Sign Up to like & getrecommendations! Published in 2021 at "Pediatric pulmonology"
DOI: 10.1002/ppul.25627
Abstract: Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are small molecules that directly impact the CFTR protein, improving the function of the CFTR chloride and bicarbonate channel. Beginning in 2012 with the Food and Drug Administration… read more here.
Keywords: modulators 2020; cystic fibrosis; cftr modulators; cftr ... See more keywords
The impact of CFTR modulators on bone health
Sign Up to like & getrecommendations! Published in 2023 at "Pediatric Pulmonology"
DOI: 10.1002/ppul.26314
Abstract: We are hopeful that CTFR modulation does have a positive impact of bone health, but we have concerns about this study design being used to support the hypothesis This article is protected by copyright. All… read more here.
Keywords: impact cftr; bone health; impact; cftr modulators ... See more keywords
Airway microbial diversity is decreased in young children with cystic fibrosis compared to healthy controls but improved with CFTR modulation
Sign Up to like & getrecommendations! Published in 2020 at "Heliyon"
DOI: 10.1016/j.heliyon.2020.e04104
Abstract: Background Culture-independent next generation sequencing has identified diverse microbial communities within the cystic fibrosis (CF) airway. The study objective was to test for differences in the upper airway microbiome of children with CF and healthy… read more here.
Keywords: cystic fibrosis; cftr modulators; diversity; healthy controls ... See more keywords
A Systematic Review of the Clinical Efficacy and Safety of CFTR Modulators in Cystic Fibrosis
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DOI: 10.1038/s41598-019-43652-2
Abstract: Several placebo-controlled trials have been recently published evaluating novel therapies targeting the defective CFTR protein. This systematic review examines the clinical efficacy and safety of CFTR modulators in individuals with cystic fibrosis (CF) with specific… read more here.
Keywords: cftr modulators; clinical efficacy; cftr; efficacy safety ... See more keywords
Discovery of CFTR modulators for the treatment of cystic fibrosis
Sign Up to like & getrecommendations! Published in 2021 at "Expert Opinion on Drug Discovery"
DOI: 10.1080/17460441.2021.1912732
Abstract: ABSTRACT Introduction Cystic fibrosis (CF) is a life-threatening inherited disease caused by mutations in the gene encoding the CF transmembrane conductance regulator (CFTR) protein, an anion channel expressed at the apical membrane of secretory epithelia.… read more here.
Keywords: cftr; cftr modulators; cystic fibrosis; discovery cftr ... See more keywords
Cystic fibrosis transmembrane conductance receptor modulator therapy in cystic fibrosis, an update.
Sign Up to like & getrecommendations! Published in 2020 at "Current Opinion in Pediatrics"
DOI: 10.1097/mop.0000000000000892
Abstract: PURPOSE OF REVIEW Cystic fibrosis transmembrane conductance receptor (CFTR) modulators are a new class of drugs that treat the underlying cause of cystic fibrosis. To date, there are four approved medications, which are mutation-specific. Although… read more here.
Keywords: modulator therapy; cystic fibrosis; fibrosis; cftr modulators ... See more keywords
To treat or not to treat: CFTR modulators after lung transplantation
Sign Up to like & getrecommendations! Published in 2021 at "Pediatric Transplantation"
DOI: 10.1111/petr.14007
Abstract: Evidence for the extrapulmonary benefits of (CFTR) modulators is rapidly expanding. The use of CFTR modulators in CF patients who have undergone lung transplantation is not clear without guidance published in the medical literature to… read more here.
Keywords: transplantation treat; transplantation; cftr modulators; lung transplantation ... See more keywords
CFTR Modulators to the Rescue of Individuals with Cystic Fibrosis and Advanced Lung Disease
Sign Up to like & getrecommendations! Published in 2021 at "American Journal of Respiratory and Critical Care Medicine"
DOI: 10.1164/rccm.202103-0674ed
Abstract: medicine in ARDS. These challenges are currently being tackled by others (https://clinicaltrials.gov/ct2/show/NCT04009330), and it is possible that we will have rapid diagnostics in the not-so-distant future. The best lesson, one that we can act on… read more here.
Keywords: medicine; cystic fibrosis; modulators rescue; rescue individuals ... See more keywords
Lived experiences of individuals with cystic fibrosis on CFTR-modulators
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DOI: 10.1186/s12890-022-01825-2
Abstract: Background CFTR-modulators are a category of drugs that facilitate trafficking and opening of the abnormal CFTR protein in individuals with cystic fibrosis (CF) who have certain genetic mutations. Clinical trial data show that individuals taking… read more here.
Keywords: cystic fibrosis; individuals cystic; cftr; lived experiences ... See more keywords
SUN-LB026 Do CFTR Modulators Treat Cystic Fibrosis-Related Diabetes?
Sign Up to like & getrecommendations! Published in 2019 at "Journal of the Endocrine Society"
DOI: 10.1210/js.2019-sun-lb026
Abstract: Abstract Background: Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are a recent advancement in the treatment of cystic fibrosis and were initially approved for their beneficial effect on lung function. There have also been few reports… read more here.
Keywords: fibrosis related; cystic fibrosis; fibrosis; related diabetes ... See more keywords
The Extrapulmonary Effects of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Cystic Fibrosis
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DOI: 10.1513/annalsats.201909-671cme
Abstract: The effects of cystic fibrosis (CF) transmembrane conductance regulator (CFTR) modulators on lung function, pulmonary exacerbations, and quality of life have been well documented. However, CF is a multiorgan disease, and therefore an evidence base… read more here.
Keywords: fibrosis transmembrane; cystic fibrosis; fibrosis; extrapulmonary effects ... See more keywords