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- W4308175317 startingPage "677" @default.
- W4308175317 abstract "Based on the cystic fibrosis transmembrane conductance regulator (CFTR) genotype, approximately 90% of people with cystic fibrosis (CF) are candidates for highly effective modulator therapy (HEMT). Clinical trials conducted over the last 11 years have shown that these oral therapies substantially restore CFTR function, leading to improvements in lung function, nutritional status, and health-related quality of life. Here, we review safety and efficacy data from phase 3 clinical trials and observational studies which support the use of HEMT in most adults and children with CF. We also discuss opportunities for additional investigation in groups underrepresented or excluded from phase 3 clinical trials, and challenges in the evaluation of the safety and efficacy of HEMT at increasingly earlier stages of CFTR-mediated pathophysiology." @default.
- W4308175317 created "2022-11-08" @default.
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- W4308175317 date "2022-12-01" @default.
- W4308175317 modified "2023-09-23" @default.
- W4308175317 title "Update on Clinical Outcomes of Highly Effective Modulator Therapy" @default.
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- W4308175317 doi "https://doi.org/10.1016/j.ccm.2022.06.009" @default.
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