Mutation-specific therapies in cystic fibrosis, 2nd Edition (PDF)

Name: Mutation-specific therapies in cystic fibrosis, 2nd Edition (PDF)
SKU: 90751
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This book provides an overview of the biology, biomarkers, and ongoing research for the development of mutation-type specific therapy of cystic fibrosis. It includes information on clinical trials, post-approval observational studies, and the real-world experience with CFTR modulators. The book is published by UNI-MED Verlag AG in its 2nd edition, is in English, and has an ISBN-10 of 3837416305 and an ISBN-13 of 978-3837416305.

Category: Medical Book

Description

Mutation-specific therapies in cystic fibrosis, 2nd Edition (PDF Book)

Cystic fibrosis is a severe ion channel disease of autosomal recessive inheritance that is caused by mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. Thanks to continuously improved symptomatic treatment during the last five decades this lethal paediatric disease has been transformed into a chronic disorder with a median life expectancy of nowadays more than 50 years. This 2nd edition provides the reader with the background and on-going preclinical and clinical research for the development of mutation-type specific therapy of cystic fibrosis. Starting with the biology and biomarkers of CFTR in the context of cystic fibrosis, the reader gets insight into the basic and clinical research of CFTR modulators from bench to bedside. A large section of the book focuses on the clinical trials, post-approval observational studies and the real-world experience with the CFTR modulators. Cystic fibrosis is a severe ion channel disease of autosomal recessive inheritance that is caused by mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. Thanks to continuously improved symptomatic treatment during the last five decades this lethal paediatric disease has been transformed into a chronic disorder with a median life expectancy of nowadays more than 50 years. This 2nd edition provides the reader with the background and on-going preclinical and clinical research for the development of mutation-type specific therapy of cystic fibrosis. Starting with the biology and biomarkers of CFTR in the context of cystic fibrosis, the reader gets insight into the basic and clinical research of CFTR modulators from bench to bedside. A large section of the book focuses on the clinical trials, post-approval observational studies and the real-world experience with the CFTR modulators.