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A Precision Medicine Approach to Optimize Modulator Therapy for Rare CFTR Folding Mutants

Trikafta, a triple-combination drug, consisting of folding correctors VX-661 (tezacaftor), VX-445 (elexacaftor) and the gating potentiator VX-770 (ivacaftor) provided unprecedented clinical benefits for patients with the most common cystic fibrosis (CF) mutation, F508del. Trikafta indications were r...

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Detalles Bibliográficos
Autores principales: Veit, Guido, Velkov, Tony, Xu, Haijin, Vadeboncoeur, Nathalie, Bilodeau, Lara, Matouk, Elias, Lukacs, Gergely L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8307171/
https://www.ncbi.nlm.nih.gov/pubmed/34357110
http://dx.doi.org/10.3390/jpm11070643