Pediatr. praxi. 2014;15(6):340-343

Therapeutic strategies in cystic fibrosis

MUDr.Veronika Skalická

Centrum cystické fibrózy

Pediatrická klinika 2. LF UK a FN Motol, Praha

Cystic fibrosis (CF) is the most common autosomal recessive disease of the Caucasian population characterised by chronic multi-organ impairment.

Although cystic fibrosis is still incurable condition, it has been shown that holistic approach to treatment that is based on multi-professional

centre care provided by trained and experienced health professionals is necessary for optimal management and improved quality of life and

survival of patients. Complex treatment focused on intensive airway clearance, good nutritional status, aggressive anti-infective and anti-inflammatory

therapy and continuous search for complications is essential. New therapeutic strategies for CF focus on protein targeted treatment

of impaired cystic fibrosis transmembrane conductance regulator (CFTR) that is responsible for exocrine secretion pathology.

Keywords: cystic fibrosis, diagnosis, standards of care, treatment, CFTR potentiators and correctors, ivacaftor

Published: December 16, 2014  Show citation