With this approval, approximately 2,250 children in the U.S. will be eligible for Trikafta® (elexacaftor/tezacaftor/ivacaftor), including more than 900 who will have access to a CFTR modulator for the first time. The Cystic Fibrosis Foundation believes it is beneficial to start people with cystic fibrosis on modulators as early as possible to help prevent lung damage and the onset of complications.
Funding will support clinical trials for therapy that could help improve digestion in people with CF
|
3 min read
Delivery of genetic therapies to affected tissues is a key challenge to developing new treatments for people with cystic fibrosis. In addition to investing in viral delivery and lipid nanoparticles, the Cystic Fibrosis Foundation is looking at the potential of a relatively new approach to delivery using an exceptionally small synthetic molecule called a polymer nanoparticle.
|
2 min read
During the annual event, volunteers advocated for the PASTEUR Act and the HELP Copays Act, two bipartisan pieces of legislation that could have substantial, positive impact on the CF community.
|
3 min read
Two-thirds of people with cystic fibrosis in the U.S. experience debt, food insecurity, or housing issues.
|
4 min read
The Foundation seeks to advance its mission by making improvements in key areas of health equity and outcomes and diverse workforce development.
|
3 min read
The Foundation’s commitment to these principles will continue to be a cornerstone of the Foundation’s work to advance our mission in 2023.
|
4 min read
The Cystic Fibrosis Foundation’s investment will go toward conducting preclinical research on a novel gene delivery vehicle. If successful, this gene delivery method could overcome some of the biggest challenges to delivering a gene therapy into the lung cells of people with cystic fibrosis.
|
2 min read