Tezacaftor
Triple therapy shows safety, tolerability for older children with cystic fibrosis
In kids aged 6 years and older with cystic fibrosis and either F508del/minimal function or F508del/F508del genotypes, elexacaftor/tezacaftor/ivacaftor was safe and well tolerated for up to 120 weeks, according to study results.
FDA approves Trikafta for children aged 2 to 5 years with cystic fibrosis
The FDA has expanded use of elexacaftor/tezacaftor/ivacaftor for children aged 2 to 5 years with cystic fibrosis with certain mutations, according to a press release from Vertex Pharmaceuticals.
Triple therapy shows safety, tolerability for young children with cystic fibrosis
Elexacaftor/tezacaftor/ivacaftor appeared safe and well tolerated for up to 24 weeks in kids aged 2 to 5 years with one or more F508del allele, according to results published in American Journal of Respiratory and Critical Care Medicine.
Some patients with cystic fibrosis may be able to stop mucoactive therapies
Discontinuing hypertonic saline or dornase alfa for 6 weeks did not appear to have a negative effect on pulmonary function among patients with cystic fibrosis on elexacaftor/tezacaftor/ivacaftor, according to study results.
Cystic fibrosis triple therapy improves lung function, early airway disease in children
Elexacaftor/tezacaftor/ivacaftor safely improved lung function and respiratory symptoms among children aged 6 to 11 years with the F508del/minimal function genotypes of cystic fibrosis, according to study results.
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.
Researcher awarded for development of triple therapy for cystic fibrosis
Marcus A. Mall, MD, whose work led to the development of the first triple therapy for patients with cystic fibrosis, won the Falling Walls Foundation’s Science Breakthrough of the Year in Life Sciences award, according to a press release.
Wearable technology shows promise in assessing physical activity, cough in cystic fibrosis
Patients receiving treatment for cystic fibrosis wore actigraphy sensors and cough monitoring systems for the majority of a 12-week period, according to a poster presented at the North American Cystic Fibrosis Conference.
COVID-19 pandemic impacted care of patients with cystic fibrosis in US
While in-person visits for patients with cystic fibrosis decreased from 2019 to 2020 during the COVID-19 pandemic, a new study found no evidence of worse lung disease, nutrition or increased risk for pulmonary exacerbations.
Cystic fibrosis therapies changing landscape of nutrition, cardiometabolic health
The use of triple therapy and other highly effective modulator therapies will likely change the landscape of nutrition and that of cardiometabolic parameters in patients with cystic fibrosis, a speaker said.