PTC Therapeutics has discontinued development of ataluren as a potential treatment for people with cystic fibrosis caused by a nonsense mutation. The Cystic Fibrosis Foundation is supporting efforts to pursue treatments for people with CF who have a nonsense, splicing or other rare mutations.
Site Search
Showing 1 - 10 of 10 results
News
|
March 2, 2009
|
1 min read
News
|
Aug. 1, 2008
|
1 min read
News
|
Aug. 11, 2014
|
2 min read
News
|
Feb. 15, 2007
|
2 min read
News
|
Aug. 11, 2014
|
1 min read
News
|
Dec. 28, 2007
|
1 min read
News
|
March 3, 2017
|
3 min read
mRNA therapy is one way to deliver the correct genetic instructions to cells, which would allow them to make functional CFTR protein regardless of an individual’s CF mutations.
3 min read
Cystic fibrosis is caused by mutations in the gene responsible for producing the cystic fibrosis transmembrane conductance regulator (CFTR) protein. For this reason, scientists are exploring ways to provide a correct copy of the gene to treat CF.
6 min read
Cystic fibrosis is caused by mutations in both copies of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Scientists are examining whether it is possible to correct the mutations through a process called gene editing.
6 min read