ORAL TREATMENT FOR CYSTIC FIBROSIS-RELATED BONE DISEASE

Abstract

Cystic fibrosis (CF) treatments have improved over the years, leading to higher life expectancies. This has made more issues related to the disease, such as cystic fibrosis-related bone disease (CFBD), more prevalent. CFBD is characterized by low bone mineral density and fragile bones. It has no known pathway or efficient treatments. Due to its role in bones, RANKL was chosen as a target for potential treatments. Small molecules were designed in an attempt to provide more leads in the search for effective treatments, especially oral, of the disease.