Experimental cystic-fibrosis treatment could be used in many diseases.

A drug that corrects the effects of a genetic mutation has produced encouraging results in tests on patients. The drug, PTC124, is designed to fool a patient’s cells into producing a functional protein, even though that protein’s gene is mutated.

Read the story here.

Experimental cystic-fibrosis treatment could be used in many diseases.

A drug that corrects the effects of a genetic mutation has produced encouraging results in tests on patients. The drug, PTC124, is designed to fool a patient’s cells into producing a functional protein, even though that protein’s gene is mutated.

Read the story here.