The revolution of personalized pharmacotherapies for cystic fibrosis: what does the future hold?

Abstract excerpt

INTRODUCTION: Cystic fibrosis (CF), a potentially fatal genetic disease, is caused by loss-of-function mutations in the gene encoding for the CFTR chloride/bicarbonate channel. Modulator drugs rescuing mutant CFTR traffic and function are now in the clinic, providing unprecedented breakthrough therapies for people with CF (PwCF) carrying specific genotypes. However, several CFTR variants are unresponsive to these...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Mutation
• Share to Molecular Targeted Therapy