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What does it all mean now that the Food and Drug Administration has issued a surprise decision to approve Sarepta Therapeutics’ Duchenne muscular dystrophy drug Vyondys 53, reversing its surprise decision to reject the drug in August?

Here are some thoughts and observations about the latest twist in the always twisty Sarepta story, including potential implications for Biogen and its controversial Alzheimer’s drug.

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  • “Eventually, Sarepta will complete confirmatory clinical trials and collect the data necessary to prove or disprove that its exon-skipping drugs — Exondys 51, Vyondys 53, and casimersen — improve muscle function in Duchenne patients.”

    You have more faith in Sarepta than I do. I don’t think they will ever willingly do those trials. They will continue to push out exon skipping drugs as fast as they can to get as much money as they can without doing any confirmatory trials until gene therapy hits the market and they can scrap the whole model.

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