Cystic Fibrosis: Understanding the Disease and Advances in Mutation-Type Specific Therapy

Cystic fibrosis is a severe ion channel disease of autosomal recessive inheritance caused by mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. Over the past five decades, improvements in treatment have transformed this once-lethal pediatric disease into a chronic disorder, with a median life expectancy now exceeding 50 years.

Background and On-Going Research

This second edition provides comprehensive insights into the ongoing preclinical and clinical research aimed at developing mutation-type specific therapy for cystic fibrosis. It delves into the biology and biomarkers of CFTR in the context of cystic fibrosis, offering readers a deep understanding of the basic and clinical research of CFTR modulators from bench to bedside.

Clinical Trials and Real-World Experience

A significant portion of the book focuses on clinical trials, post-approval observational studies, and real-world experience with CFTR modulators. This section provides valuable information on the efficacy, safety, and practical implications of these treatments, shedding light on the future prospects for cystic fibrosis patients.

By exploring the latest advances in cystic fibrosis research and treatment, this book serves as a valuable resource for healthcare professionals, researchers, and individuals affected by the disease, offering hope for a brighter future through the development of effective, mutation-type specific therapies.