Development of Cystic Fibrosis Transmembrane Regulator Therapies

Patricia L Darbishire

doi:10.4172/2329-6631.1000e117

Abstract

Do you know someone with cystic fibrosis? I did not, until a lovely young college student in her twenties asked for assistance researching the latest advances in cystic fibrosis (CF) treatments. She confided her interest to me in new drug development. The average life expectancy for someone with CF is under 40 years of age. Thus began our journey into a review of conventional CF therapies and new drug entities in the investigational pipeline. Working alongside my newfound friend has propelled my desire to educate my own pharmacy students, as well as fellow clinicians, researchers and the general public, on advances in CF care. Recent innovations in drug development hold promise in targeting the underlying cause of CF. We believe my friend now has hope for a long, bright future.

Highlights

Do you know someone with cystic fibrosis? I did not, until a lovely young college student in her twenties asked for assistance researching the latest advances in cystic fibrosis (CF) treatments
Disease manifestations are due to a mutation of the cystic fibrosis transmembrane regulator (CFTR) gene located on the seventh chromosome
All mutations result in functional impairment of the CFTR protein which serves as a chloride channel on epithelial cells

Summary

Introduction

Do you know someone with cystic fibrosis? I did not, until a lovely young college student in her twenties asked for assistance researching the latest advances in cystic fibrosis (CF) treatments. Development of Cystic Fibrosis Transmembrane Regulator Therapies Recent innovations in drug development hold promise in targeting the underlying cause of CF. Disease manifestations are due to a mutation of the cystic fibrosis transmembrane regulator (CFTR) gene located on the seventh chromosome.

Results

Conclusion