J Barben, KW Southern - International journal of neonatal screening, 2020 - mdpi.com
The introduction and widespread implementation of newborn bloodspot screening (NBS) for cystic fibrosis (CF) has offered earlier diagnosis and better outcomes for children with CF in …
In the not-too-distant past, parents were counseled that children with cystic fibrosis were unlikely to live past their teenage years. But a new estimate of patients' life spans suggests …
V Terlizzi, PM Farrell - Current Problems in Pediatric and Adolescent …, 2024 - Elsevier
During the past quarter century, the diagnosis and treatment of cystic fibrosis (CF) have been transformed by molecular sciences that initiated a new era with discovery of the cystic …
SM Paranjape, PJ Mogayzel Jr - Paediatric respiratory reviews, 2018 - Elsevier
The treatment of people with cystic fibrosis (CF) has been transformed by the availability of drugs that target the basic chloride defect in the disease. The use of drugs that target specific …
SL Martiniano, JE Hoppe, SD Sagel… - Advances in …, 2014 - advancesinpediatrics.com
INTRODUCTION Cystic fibrosis (CF) is a genetic disorder that leads to chronic multisystem disease consisting of chronic sinopulmonary infections, malabsorption, and nutritional …
The first regulatory approval for a drug developed specifically for cystic fibrosis (CF) occurred in 1993, and since then, several other drugs have been approved. Median …
J Massie, PJ Robinson… - Journal of Paediatrics and …, 2016 - Wiley Online Library
There have been dramatic changes in the care of children with cystic fibrosis (CF) from 1965 to 2015. The initial improvements were the result of incremental gains in medical knowledge …
C Castellani, B Linnane, I Pranke… - … in respiratory and …, 2019 - thieme-connect.com
The diagnosis of cystic fibrosis (CF) has traditionally relied on the presence of clinical features of the disease. Today, diagnosis through newborn screening (NBS) is becoming the …
SL Martiniano, SD Sagel… - Current opinion in …, 2016 - journals.lww.com
CFTR modulators promise to transform the therapeutic landscape in CF in a precision based fashion. Areas of ongoing research include developing drugs for all mutation classes so that …