Casting Doubts On The Molecular Mechanism Of 'Read-Through' Drug PTC124/Ataluren

A drug developed to treat genetic diseases such as Duchenne muscular dystrophy and cystic fibrosis may need a radical rethink. In a new study published in the open access journal PLOS Biology, researchers question the mechanistic basis of the drug called PTC124 (also known as Ataluren), casting doubt as to whether it has the molecular effects that are claimed for it. This may have implications for its effectiveness in treating genetic diseases. An estimated 10% of all human genetic diseases are caused by nonsense mutations...
Source: Health News from Medical News Today - Category: Consumer Health News Tags: Muscular Dystrophy / ALS Source Type: news