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Idil Apak Evans on Muck Rack

Idil Apak Evans

As seen in: The Journal of Clinical Investigation
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Articles

In utero and postnatal ivacaftor/lumacaftor therapy rescues multiorgan disease in CFTR-F508del ferrets

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By JCI Insight, Xingshen Sun, Idil Apak Evans, Amber R. Vegter
| The Journal of Clinical Investigation
Abstract Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator (CFTR) gene, with F508del being the most prevalent mutation. The combination of CFTR modulators (potentiator and correctors) has provided benefit to CF patients carrying the F508del mutation; however, the safety and effectiveness of in utero combination modulator therapy remains unclear.
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