Society for Pediatric Research Presidential Address 2011: improving child health outcomes—from 65 roses to high reliability

Novel personalized therapies for cystic fibrosis: treating the basic defect in all patients

“…Despite the apparent ‘simplicity’ of being monogenic (and ‘simplification’ of the name to ‘65 roses’ by children with CF ), CF still poses many diagnostic challenges and its clinical management is not straightforward. CF is in fact a complex disorder for several reasons.…”