On Dec. 18, the Fifth Circuit Court of the United States issued a decision in Texas v. United States, ruling the individual mandate unconstitutional but sending the case back down to the district court for further review about the fate of the entire Affordable Care Act.
Ray Poole and Julie Riedy to lead efforts in Washington, D.C., for volunteer conference, Until CF Stands for Cure Found.
Nonprofit issues challenge to accelerate treatments for every person with CF
The Cystic Fibrosis Foundation announced today that it has licensed a compound to the biopharmaceutical company AbbVie to develop into a potential CFTR modulator treatment.
The U.S. Food and Drug Administration has approved the use of the triple-combination modulator elexacaftor/tezacaftor/ivacaftor (Trikafta™) for people with cystic fibrosis ages 12 and older who have at least one copy of the F508del mutation.
This medicine represents the single greatest therapeutic advancement in the history of CF, offering a treatment for the underlying cause of the disease that could eventually benefit more than 90 percent of people with CF.
The Cystic Fibrosis Foundation has awarded up to $7 million to Enterprise Therapeutics to develop a compound that targets a non-CFTR chloride channel in lung cells. If successful, the drug could help mucus become more hydrated and easier to clear from the lungs of all people with CF, regardless of their CFTR mutations.
The Institute for Clinical and Economic Review (ICER) recently posted a draft scoping document to outline their planned assessment of the clinical effectiveness and value of the triple combination (also known as elexacaftor/tezacaftor/ivacaftor). The document is open for public comment through October 21.
The Cystic Fibrosis Foundation awarded up to $1.61 million to Eloxx Pharmaceuticals Inc. to conduct a U.S.-based Phase 2 clinical trial of an investigational drug that could potentially treat people with cystic fibrosis who have a nonsense mutation.
The U.S. Food and Drug Administration has approved the use of tezacaftor/ivacaftor (Symdeko®) for children with cystic fibrosis ages 6 to 11 with specific mutations.