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Recently-developed modulators for the cystic fibrosis transmembrane conductance regulator (CFTR) were approved by the US Food and Drug Administration for initial use in 2012.1 Major impacts from this emerging pharmacotherapy have improved nutritional status and lung function and better quality of life.2 Further, these groundbreaking CFTR modulators are expected to increase life expectancy beyond the current median US survival of 41 years.3 This will be accompanied by significant chronic symptoms and recognition of new health issues along the way. These new challenges highlight the importance of individualised palliative treatment plans to remedy existing and emerging gaps in care.
Over the past decade, improvements in CF treatment have changed the face of the disease. While the current focus rightfully lies on the positive outcomes associated with CFTR modulators, redefining the disease has consequences. Moreover, increased survival into adulthood ushers in …
Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.
Competing interests None declared.
Patient consent for publication Not required.
Provenance and peer review Not commissioned; internally peer reviewed.
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