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Article

PTI-801 (posenacaftor) shares a common mechanism with VX-445 (elexacaftor) to rescue p.Phe508del-CFTR.

European journal of pharmacology - 15 Mar 2024

Ferreira Filipa C, Amaral Margarida D, Bacalhau Mafalda, Lopes-Pacheco Miquéias

Abstract excerpt

The deletion of a phenylalanine at position 508 (p.Phe508del) in the CFTR anion channel is the most prevalent variant in people with Cystic Fibrosis (CF). This variant impairs folding and stability of the CF transmembrane conductance regulator (CFTR) protein, resulting in its defective trafficking and premature degradation. Over the last years, therapeutic accomplishments have been attained in developing small...

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PMID: 38336013
DOI: 10.1016/j.ejphar.2024.176390

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PTI-801 (posenacaftor) shares a common mechanism with VX-445 (elexacaftor) to rescue p.Phe508del-CFTR.PMID 38336013Read ↗

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