Article
Managing cystic fibrosis in children aged 6-11yrs: a critical review of elexacaftor/tezacaftor/ivacaftor combination therapy.
Expert review of respiratory medicine - 1 Feb 2023
Jordan Kamyron D, Zemanick Edith T, Taylor-Cousar Jennifer L, Hoppe Jordana E
Abstract excerpt
INTRODUCTION: Cystic fibrosis is a life-limiting, autosomal recessive genetic disorder resulting in multi-organ disease due to CF transmembrane conductance regulator (CFTR) protein dysfunction. CF treatment previously focused on mitigation of disease signs and symptoms. The recent introduction of highly effective CFTR modulators, for which ~90% of people with CF are CFTR variant-eligible, has resulted in...
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