Cystic Fibrosis Transmembrane Conductance Regulator
Vanzacaftor, tezacaftor, deutivacaftor safe in children with cystic fibrosis
Patients aged 6 to 11 years with cystic fibrosis receiving 24-week once-daily vanzacaftor/tezacaftor/deutivacaftor generally tolerated it well, according to data published in The Lancet Respiratory Medicine.
Vanzacaftor, tezacaftor, deutivacaftor similar to current cystic fibrosis standard of care
Among individuals aged at least 12 years with cystic fibrosis, receipt of vanzacaftor/tezacaftor/deutivacaftor was similar or better than Trikafta depending on the endpoint, according to results published in The Lancet Respiratory Medicine.
FDA approves Alyftrek for cystic fibrosis treatment
The FDA has approved Alyftrek for patients aged 6 years and older with cystic fibrosis with certain mutations, according to a press release from Vertex Pharmaceuticals.
Cystic fibrosis triple therapy improves clinical outcomes in N1303K mutation
Percent-predicted FEV1 and respiratory-related quality of life improved with 28-day triple therapy in individuals with the N1303K cystic fibrosis transmembrane conductance defect, according to data published in Lancet Respiratory Medicine.
Cystic fibrosis triple therapy outcomes improve regardless of prior modulators
BOSTON — Having vs. not having past cystic fibrosis transmembrane conductance regulator modulator exposure did not change positive outcomes seen with elexacaftor/tezacaftor/ivacaftor, according to data presented at the CHEST Annual Meeting.
Q&A: Finding a treatment for rare cystic fibrosis splicing mutation
Although inhaled antisense oligonucleotide treatment is safe, its efficacy needs to be determined in patients with the cystic fibrosis splicing mutation 3849+10kb C-to-T, according to a press release from the Cystic Fibrosis Foundation.
Spotting a Zebra: When to Suspect Cystic Fibrosis and What to Do Next
This activity is supported by an independent educational grant from Vertex Pharmaceuticals.
Q&A: Zinc transport protein may help decrease bacterial infections in cystic fibrosis
Patients with cystic fibrosis frequently experience bacterial infections, but a zinc transport protein may be able to lower infection incidence, according to findings published in Proceedings of the National Academy of Sciences.
Yearlong gap in care linked to decreased lung function in cystic fibrosis
Patients with cystic fibrosis had reduced lung function if they had at least one 12-month or longer gap in receiving care, according to findings published in Annals of the American Thoracic Society.
Q&A: Genetic technologies aim to advance cystic fibrosis treatments
In patients with cystic fibrosis, a new genetic technology could permanently fix the CF transmembrane conductance regulator gene in hundreds of disease-causing CF mutations, according to a press release from the Cystic Fibrosis Foundation.