Article

INTEGRATING GENOMIC AND FUNCTIONAL TESTING TO IMPROVE CFTR MODULATOR RESPONSE PREDICTION IN CHILDREN WITH CYSTIC FIBROSIS

2025-07-11

Abstract excerpt

<h4>ABSTRACT</h4> <h4>Background</h4> CFTR modulators have transformed cystic fibrosis (CF) treatment, but individual responses vary even among patients with identical CFTR genotypes. This underscores the need for predictive biomarkers to optimize therapeutic selection. <h4>Methods</h4> We evaluated 24 paediatric patients homozygous for F508del-CFTR, assessing lung function (FEV1pp) and sweat chloride (SC) befo...

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Literature Corpus work: db3eef1e-2d47-5617-bc62-262d31627c52
DOI: 10.1101/2025.07.09.25331152

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