A survey: Understanding the health and perspectives of people with CF not benefiting from CFTR modulators

Background While the advent of cystic fibrosis transmembrane conductance regulator (CFTR) modulator use has improved daily life and long‐term prognosis of CF for many with approved CFTR mutations, approximately 10% of people with CF (pwCF) have only symptomatic treatments available. Methods Between...

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Veröffentlicht in:	Pediatric pulmonology 2022-05, Vol.57 (5), p.1253-1261
Hauptverfasser:	Kramer‐Golinkoff, Emily, Camacho, Amanda, Kramer, Liza, Taylor‐Cousar, Jennifer L.
Format:	Artikel
Sprache:	eng
Schlagworte:	10 CFTR modulator Clinical trials Cystic fibrosis Cystic Fibrosis - drug therapy Cystic Fibrosis - genetics Cystic Fibrosis Transmembrane Conductance Regulator - genetics Cystic Fibrosis Transmembrane Conductance Regulator - therapeutic use Humans Mutation nonsense mutation Original ORIGINAL ARTICLES orphan disease Surveys and Questionnaires
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