Effectiveness of lumacaftor/ivacaftor initiation in children with cystic fibrosis aged 2 through 5 years on disease progression: Interim results from an ongoing registry-based study

Cystic fibrosis (CF), an autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, affects an estimated 105,000 people worldwide, nearly half being children (under the age of 18 years) [1,2]. Clinical manifestations of CF begin early in life and include progressive structural lung damage and pancreatic insufficiency [3 –5]. In the lungs, mucus hyperconcentration and decreased mucociliary clearance can lead to air flow obstruction, infection, and inflammation and eventually the development of severe bronchiectasis and respiratory failure [6], while pancreatic damage caused by CF can result in nutrient malabsorpti on and poor weight gain [5].
Source: Journal of Cystic Fibrosis - Category: Respiratory Medicine Authors: Tags: Original Article Source Type: research