The Institute for Clinical and Economic Review (ICER) is developing an assessment of the clinical effectiveness and value of the triple combination (also known as elexacaftor/tezacaftor/ivacaftor). The first opportunity to comment runs through Sept. 25.
The Cystic Fibrosis Foundation has increased its award to Arcturus Therapeutics to up to $15 million to develop a mutation-agnostic therapy for people with cystic fibrosis.
To support a pair of colleagues' adoption of a baby boy, Christian, Merrill/Bank of America has raised more than $32,000 through their matching gifts program.
Recipients include five innovative programs from around the country that empower the CF community.
Vertex Pharmaceuticals Inc. announced today that it submitted a New Drug Application to the U.S. Food and Drug Administration for the approval of the first triple-combination therapy for cystic fibrosis.
U.S. Rep. Mark Amodei of Nevada will join fellow Rep. Jim McGovern and Sens. Chuck Grassley and Ed Markey in co-chairing the Congressional Cystic Fibrosis Caucus, championing awareness of the rare disease and the Cystic Fibrosis Foundation's search for a cure.
Nearly 200 people from across the country -- including teens from 38 states -- came to Washington, D.C., on June 27 to advocate for the needs of their loved ones living with cystic fibrosis during the Cystic Fibrosis Foundation's 11th Annual Teen Advocacy Day.
Vertex Pharmaceuticals Inc. today announced that it will pursue FDA approval for the triple-combination therapy VX-445 (elexacaftor) plus tezacaftor/ivacaftor (Symdeko®).
Preston W. Campbell, III, President and CEO to retire; Michael P. Boyle, MD, appointed as successor effective January 2020.
The U.S. Food and Drug Administration has approved the use of ivacaftor (Kalydeco®) for children as young as 6 months.