New study found that experimental drug called PTC124 which is effective for muscular dystrophy treatment can also help to treat cystic fibrosis. It seems that this drug has ability to rescue faulty proteins that lead to illness and researchers hope to finding treatment for more than two thousand diseases that are caused by certain class of DNA mutation.

During the research on mice team found that the drug was able to restore up to 30% of the abnormal cystic – fibrosis protein cases to normal function. This study has shown that this drug is capable of suppressing certain mutations that cause cystic fibrosis in the first place and researchers hope to find in following trials what kind of effect PTC124 has on mutation in cystic fibrosis patients.

It was discovered that in CF patients, lack of certain protein leads to imbalance of salt and water in the linings of the lungs and this drug allowed protein to be made in mouse cells where it was previously absent. This drug was able to restore salt and water balance in membranes. Researchers hope this drug will be of great importance for finding new therapies for cystic fibrosis.