51 Orkambi: the expectations of benefit for people in the UK with cystic fibrosis

The psychological implications and health risks of cystic fibrosis pre- and post- CFTR modulator therapy

“…In 2019, modulator therapies became available in the UK via the NHS, with these drugs targeting the underlying cause of CF by treating and correcting specific genetic mutations, and are genomespecific. [11][12][13] Currently, modulator therapies (Kalydeco, Orkambi, Symkevi and Kaftrio) are available for 90% of the UK CF population, 14 however, quality-of-life, reformulations of identity and mental health have not been explored in this new era of CF care. Anecdotally individuals with CF today are reporting the changing effects of modulator drugs on physical health and quality-of-life, with reports of dramatic increases in lung function, weight gain, and the ability to participate in previously inaccessible activities.…”