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Toward inclusive therapy with CFTR modulators: Progress and challenges

Cystic fibrosis is caused by gene mutations that result in an abnormal Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein on the surface of cells. CFTR modulators are a novel class of drugs that directly target the molecular defect. CFTR modulators include potentiators that result in...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Veröffentlicht in:Pediatr Pulmonol
Hauptverfasser: Guimbellot, Jennifer, Sharma, Jyoti, Rowe, Steven M.
Format: Artigo
Sprache:Inglês
Veröffentlicht: 2017
Schlagworte:
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6208153/
https://ncbi.nlm.nih.gov/pubmed/28881097
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ppul.23773
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