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Prediction of Real-World Long-Term Outcomes of People with CF Homozygous for the F508del Mutation Treated with CFTR Modulators
The clinical response to cystic fibrosis transmembrane conductance regulator (CFTR) modulators is variable within people with cystic fibrosis (pwCF) homozygous for the F508del mutation. The prediction of clinical effect in individual patients would be useful to target therapy to those who would bene...
Autores principales: | Muilwijk, Danya, Bierlaagh, Marlou, van Mourik, Peter, Kraaijkamp, Jasmijn, van der Meer, Renske, van den Bor, Rutger, Heijerman, Harry, Eijkemans, René, Beekman, Jeffrey, van der Ent, Kors |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8707616/ https://www.ncbi.nlm.nih.gov/pubmed/34945848 http://dx.doi.org/10.3390/jpm11121376 |
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