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Drs. Jennifer Goralski and George Retsch Bogart

Investigators in Pediatric Pulmonology at UNC Children’s are playing key roles in the study and treatment of Cystic Fibrosis (CF). Cystic Fibrosis is a progressive, genetic disease that affects the lungs, pancreas, and other organs. In the United States, nearly 40,000 children and adults, from every racial and ethnic group, are living with cystic fibrosis, with approximately 900 new cases diagnosed annually. Almost 1,000 North Carolinians have been diagnosed with this life-threatening condition.

Since 2009, CF has been part of newborn screening in North Carolina. Earlier detection means children can be seen by physicians sooner, every 2 months for their first year of life and every 3 months thereafter, allowing for the best possible nutritional care, monitoring, and treatment of respiratory issues. Since its implementation, the screening test in North Carolina has identified between 30 and 34 newborns with CF each year. Though there is no cure for CF, newborn screening and ongoing research means, immediately after diagnosis, children can begin treatment on developing therapies that dramatically change their lives.

According to the Cystic Fibrosis Foundation, the current life expectancy for CF patients born between 2017 and 2021 is 53 years — a substantial change from just 10 years ago when the life expectancy was 38. These incredible improvements in the lives of children and adults with CF have been possible through years of laboratory and translational research at UNC and medical centers around the world. At UNC Children’s, clinical trials that test potential drugs and therapies in people with CF are a fundamental part of CF research and care.

Faculty members in UNC Pediatric Pulmonology play ongoing, key roles in studying multiple aspects of CF including the application of highly-effective cystic fibrosis transmembrane conductance regulator (CFTR) in the care and treatment of children with cystic fibrosis. These are life-changing therapies and a shining example of successful clinical trials in young children. The publications below by Dr. Jennifer Goralski and Dr. George Retsch-Bogart reflect the strong work being done by everyone at UNC to improve the lives of children and adults with CF in North Carolina and around the world.

American Journal of Respiratory and Critical Care Medicine Volume 208, Issue 1| July 1 2023