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

Alejandro Teper,Silvina Lubovich,Viviana Rodríguez, Silvina Zaragoza, Ezequiel Rodríguez, Facundo García Bournissen

Journal of Cystic Fibrosis（2023）

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摘要

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.

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cystic fibrosis,cftr

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