Please provide your email address to receive an email when new articles are posted on . Patients given aerosolized 4D-710 had elevated CFTR protein levels that went over normal levels. Over 12 months, ...
Researchers have found in a new study that triple CFTR modulator therapy, or elexacaftor/tezacaftor/ivacaftor (ETI), produces ...
“This Phase 2a trial evaluated the potential effect of an oral compound to improve trafficking of the defective CFTR protein, and its results represent an encouraging step forward in the development ...
Purpose: Most cystic fibrosis mutation screening methods do not detect large exon deletions or duplications in the cystic fibrosis transmembrane regulator gene. We looked for such mutations in ...
The CFTR protein is expressed in epithelial cells, where it is involved in the transport of chloride ions. Since the identification of the CFTR gene, over 1500 different mutations have been identified ...
Children ages two to five who have the most common form of cystic fibrosis (CF), caused by two copies of the F508 gene mutation, have not had any modulator treatments available to them until recently.
Care for patients with cystic fibrosis has undergone transformative changes over the past decade and serves as an example of how an understanding of the functional consequences of a genetic disease ...
A study published in Nature Communications revealed a new antisense oligonucleotide (ASO) therapy applicable to the W1282X mutation of the cystic fibrosis transmembrane conductance regulator gene ...
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