Organoid-guided synergistic treatment of minimal function CFTR mutations with CFTR modulators, roflumilast and simvastatin: a personalised approach

Abstract excerpt

Highly effective cystic fibrosis transmembrane conductance regulator (CFTR) protein-targeting modulator therapies (HEMTs) facilitate strong clinical improvements in a large proportion of people with CF (PwCF) [1, 2]. More specifically, the EMA and FDA approved combination of the CFTR modulators elexacaftor/tezacaftor/ivacaftor (ETI) for PwCF with at least one F508del allele, whilst the FDA extended eligibility...

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