Cystic fibrosis: current therapeutic targets and future approaches.

ABSTRACT:

Objectives

Study of currently approved drugs and exploration of future clinical development pipeline therapeutics for cystic fibrosis, and possible limitations in their use.

Methods

Extensive literature search using individual and a combination of key words related to cystic fibrosis therapeutics.

Key findings

Cystic fibrosis is an autosomal recessive disorder due to mutations in CFTR gene leading to abnormality of chloride channels in mucus and sweat producing cells. Respiratory system and GIT are primarily involved but eventually multiple organs are affected leading to life threatening complications. Management requires drug therapy, extensive physiotherapy and nutritional support. Previously, the focus was on symptomatic improvement and complication prevention but

SUBMITTER: Rafeeq MM 

PROVIDER: S-EPMC5408469 | biostudies-literature | 2017 Apr

REPOSITORIES: biostudies-literature