Target: Mutation

The US Food and Drug Administration has approved "the first therapy to treat an underlying cause of cystic fibrosis, a pill that targets a genetic mutation affecting a small minority of patients suffering from the life-threatening breathing disorder," The Wall Street Journal reports. Vertex Pharmaceuticals' Kalydeco targets G551D, a mutation in the cystic fibrosis transmembrane conductance regulator, or CFTR, gene.

To read the full story....

Register for Free.

Already have a GenomeWeb or 360Dx account?
Login Now.

What happens to scientific papers when certain journals are no longer published? Some scientists are trying to make sure they don't disappear forever.

A study in Microbiome finds that heavy drinkers have an unhealthy mix of bacteria in their mouths.

Doctors and patients are still trying to figure out what role at-home genetic testing should play in healthcare, Newsweek says.

In Genome Research this week, mismatch repair deficiency in C. elegans, retracing transcriptions start site evolution in the human genome, and more.