New drugs, new challenges in cystic fibrosis care.

Abstract excerpt

Cystic fibrosis (CF) is a genetic disease caused by variants in the gene encoding for the CF transmembrane conductance regulator (CFTR) protein, a chloride and bicarbonate channel. CFTR dysfunction results in a multiorgan disease with the main clinical features being exocrine pancreatic insufficiency and diffuse bronchiectasis with chronic airway infection leading to respiratory failure and premature death. Over...

Topics

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• Cystic Fibrosis
• Cystic Fibrosis Transmembrane Conductance Regulator
• Genetic Predisposition to Disease
• Lung
• Molecular Targeted Therapy