Cystic Fibrosis Transmembrane Conductance Regulator
FDA grants orphan drug designation to cystic fibrosis treatment
The FDA granted orphan drug designation to ARCT-032 as a treatment for patients with cystic fibrosis, according to a press release from Arcturus Therapeutics.
Aerosol gene therapy safe, heightens CFTR protein levels in cystic fibrosis
Receipt of 4D-710, an aerosolized gene therapy, resulted in high cystic fibrosis transmembrane conductance regulator protein expression levels in patients with cystic fibrosis, according to a company press release.
‘CFTR dysfunction may be a lot more common’ in healthy young adults
WASHINGTON — Among healthy young adults, 28% had intermediate sweat chloride levels, with lung function measures similar to those with normal levels, according to a poster presented at the American Thoracic Society International Conference.
Novel biomarker shows promise in assessing CFTR function in patients with cystic fibrosis
Challenged urine bicarbonate excretion provided a simple and safe quantification of cystic fibrosis transmembrane conductance regulator function among adults with cystic fibrosis, according to data published in Annals of Internal Medicine.
FDA approves expanded use of three cystic fibrosis therapies
The FDA has approved expanded indications for three medications for the treatment of patients with cystic fibrosis with specific mutations in the CFTR gene, according to press release from Vertex Pharmaceuticals.
Outcomes better with triple therapy in cystic fibrosis with F508del mutation
When compared with treatment with tezacaftor plus ivacaftor alone, triple combination therapy with elexacaftor/tezacaftor/ivacaftor improved lung function, respiratory-related quality of life and sweat chloride concentrations in patients aged 12 years and older with cystic fibrosis homozygous for the F508del mutation, according to data published in The Lancet.
Triple therapy safe, effective in cystic fibrosis with single Phe508del allele
Combination therapy with elexacaftor/tezacaftor/ivacaftor improved lung function and the rate of pulmonary exacerbations with minimal adverse effects in patients aged 12 years and older with cystic fibrosis with Phe508del-minimal function genotypes who did not previously respond to cystic fibrosis transmembrane conductance regulator modulator therapy, according to a study published in The New England Journal of Medicine.