FDA declines to approve PTC's Duchenne drug

Reuters – The U.S. Food and Drug Administration has declined to approve PTC Therapeutics Inc’s experimental drug to treat Duchenne muscular dystrophy, saying an additional clinical trial would be needed to prove the drug works.

The company said on Wednesday that it strongly disagreed with the agency’s conclusions and plans to file a formal dispute next week. The drug, ataluren, is designed to treat Duchenne muscular dystrophy (DMD) caused by a mutation in the DMD gene known as a nonsense mutation.

Read more at Reuters.


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