Cystic Fibrosis: The Year in Review

Insights into the underlying causes and potential treatment options for cystic fibrosis continued to move forward in 2017. In July of this year, Vertex Pharmaceuticals made a big announcement regarding a drug trail they’d been conducting. The company had been conducting early stage clinical trials studying the effect that next generation modulators can have on the underlying cause of cystic fibrosis in individuals with a single F508del CFTR mutation. Essentially, the trial uses combinations of three different compounds to treat the cell level mutations of one F508del CFTR and minimal function cells.