A survey: Understanding the health and perspectives of people with CF not benefiting from CFTR modulators.
Emily Kramer-GolinkoffAmanda CamachoLiza KramerJennifer L Taylor-CousarPublished in: Pediatric pulmonology (2022)
PwCF who are ineligible, intolerant, or lack access to modulators have a high burden of disease impacting their physical and mental health. Although most are happy for those who are benefiting from modulators, they are eager for the opportunity to experience similar improvements for themselves, and willing to participate in clinical trials of new therapies.