News

Scientists Hope They Have Discovered New Form of CF Therapy

Scientists have discovered a new way to treat cystic fibrosis (CF) that involves delivering artificial proteins to patients’ lung cells to replace the faulty cystic fibrosis transmembrane conductance regulator (CFTR) protein. The finding was reported in the study, “Anion carriers as potential treatments for cystic fibrosis: transport in…

Vertex Partners with Ribometrix to Discover RNA-targeted Therapies

Vertex Pharmaceuticals has entered a strategic collaboration with Ribometrix to discover potential RNA-targeted therapies for serious diseases. Vertex is a leader in the market of CFTR modulators for cystic fibrosis (CF), and is the developer of Kalydeco (ivacaftor), Orkambi (ivacaftor/lumacaftor), and Symdeko (tezacaftor/ivacaftor combination therapy), three approved CF…

NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC

Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…