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CFTR Modulators for the Treatment of Cystic Fibrosis

Defects in a single gene lead to the defective proteins that cause cystic fibrosis, making the disease an ideal candidate for mutation-targeted therapy. Although ivacaftor is currently the only FDA-approved CFTR modifier, others are in development.

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Detalhes bibliográficos
Main Authors: Pettit, Rebecca S., Fellner, Chris
Formato: Artigo
Idioma:Inglês
Publicado em: MediMedia USA, Inc. 2014
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4103577/
https://ncbi.nlm.nih.gov/pubmed/25083129
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