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New insights into structure and function of bis-phosphinic acid derivatives and implications for CFTR modulation

Archive ouverte | Bitam, Sara | CCSD

International audience. C407 is a compound that corrects the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein carrying the p.Phe508del (F508del) mutation. We investigated the corrector effect of c4...

Systemic bis-phosphinic acid derivative restores chloride transport in Cystic Fibrosis mice

Archive ouverte | da Cunha, Mélanie Faria | CCSD

International audience. Mutations in the Cystic Fibrosis Transmembrane Conductance Regulator gene (CFTR) are responsible for Cystic Fibrosis (CF). The most common CF-causing mutation is the deletion of the 508th ami...

Theratyping cystic fibrosis patients to guide elexacaftor/tezacaftor/ivacaftor out-of-label prescription

Archive ouverte | Dreano, Elise | CCSD

International audience. Background Around 20% of people with cystic fibrosis (pwCF) do not have access to the triple combination elexacaftor/tezacaftor/ivacaftor (ETI) in Europe because they do not carry the F508del...

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