TY - JOUR T1 - Modulator treatment for people with cystic fibrosis: moving in the right direction JF - European Respiratory Review JO - EUROPEAN RESPIRATORY REVIEW DO - 10.1183/16000617.0051-2020 VL - 29 IS - 155 SP - 200051 AU - J. Stuart Elborn Y1 - 2020/03/31 UR - http://err.ersjournals.com/content/29/155/200051.abstract N2 - In the past 10 years the incremental success of modulators of the cystic fibrosis transmembrane regulator (CFTR) protein have improved outcomes for people with cystic fibrosis (CF) [1]. Ivacaftor for individuals with sequence variants associated with class III mutations (e.g. G551D) and some other residual function mutations of the CFTR gene followed by lumacaftor/ivacaftor or tezacaftor/ivacaftor combinations in individuals homozygous for the F508del have achieved improvements in quality of life reduction, pulmonary exacerbations and variable improvement in forced expiratory volume in 1 s (FEV1). Highly effective modulator treatment, a triple combination of tezacaftor/ivacaftor and elexacaftor (a second modulator with the CFTR potentiator), was granted market approval in October 2019 by the US Food and Drug Administration. This approval is “for patients 12 years and older with CF who have at least one F508del mutation in the CFTR gene, which is estimated to represent 90% of the CF population” [2].As delivery of CF healthcare services evolves, it is important for respiratory clinical teams and the wider group of specialists in the MDT to understand how modulator drugs are used, the side-effects and potential for drug interactions http://bit.ly/3akQ0tK ER -