Real-life experience with a generic formulation of lumacaftor-ivacaftor in patients with cystic fibrosis homozygous for the Phe508del CFTR mutation.

Alejandro TeperSilvina LubovichViviana RodríguezSilvina ZaragozaEzequiel RodríguezFacundo García Bournissen

Published in: Pediatric pulmonology (2023)

The use of a generic formulation of lumacaftor-ivacaftor in patients homozygous for the Phe508del CFTR mutation was associated with improvement in nutritional status and respiratory symptoms, and a significant reduction in severe pulmonary exacerbations.

Keyphrases

cystic fibrosis
end stage renal disease
newly diagnosed
ejection fraction
chronic kidney disease
peritoneal dialysis
drug delivery
prognostic factors
chronic obstructive pulmonary disease
pulmonary hypertension
early onset
sleep quality