E. Ukor
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View article: The effectiveness of CFTR modulators in people with CF and rare mutations: A real‐world study
The effectiveness of CFTR modulators in people with CF and rare mutations: A real‐world study Open
Cystic fibrosis (CF) is an autosomal recessive disease, caused by reduced function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. CFTR modulators (CFTRm) directly target this defect, first becoming commercially …