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Sarepta Outlines A Fast Path Forward For Its DMD Gene Therapy

 
• By Jessica Merrill

Encouraging data testing the gene therapy in just three boys with Duchenne muscular dystrophy has investors jazzed. CEO Doug Ingram said the company will talk to FDA about how to advance the program. 

genetic reasearch

Sarepta Therapeutics Inc. is poised to get a second chance to deliver a treatment to children with Duchenne muscular dystrophy, this time perhaps with a stronger efficacy result. The company unveiled the first data on a new gene therapy in three boys that could represent a breakthrough in the treatment of the debilitating disease – if the results can hold through to a broader patient population.

It’s a familiar situation for the DMD pioneer, which eventually gained approval from the US FDA for the first DMD therapy, Exondys 51 (eteplirsen), after generating strong early evidence and...

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