Article
New treatments targeting the basic defects in cystic fibrosis.
Presse medicale (Paris, France : 1983) - 1 Jun 2017
Fajac Isabelle, Wainwright Claire E
Abstract excerpt
Cystic fibrosis (CF) is a monogenic autosomal recessive disorder affecting around 75,000 individuals worldwide. It is a multi-system disease but the main morbidity and mortality is caused by chronic lung disease. Due to newborn screening, a multidisciplinary approach to care and intensive symptomatic treatment, the prognosis has dramatically improved over the last decades and there are currently more adults than...
Topics
- Adolescent
- Adult
- Aminophenols
- Child
- Child, Preschool
- Chloride Channel Agonists
- Cystic Fibrosis
- Cystic Fibrosis Transmembrane Conductance Regulator
- DNA Mutational Analysis
- Exocrine Pancreatic Insufficiency
- Genetic Therapy