Methods and therapeutic compositions for treating cystic fibrosis

摘要

Methods for treating cystic fibrosis are described. The methods involve the administration of a protein enhancing agent, differentiating agent and/or carboxy-compound to a subject afflicted with cystic fibrosis such that mutant cystic fibrosis transmembrane regulator protein present within cystic fibrosis-associated cells becomes functional. Other aspects described include therapeutic compositions and packaged drugs.