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Vyondys 53 Clinical Development Timeline

Executive Summary

Chronicle of the development and review of Sarepta’s golodirsen for treatment of Duchenne muscular dystrophy patients with a genetic mutation amenable to exon 53 skipping.

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With Sarepta’s Vyondys, US FDA Staff Saw Opportunity To Right Perceived Wrongs With Exondys

ODE I director Ellis Unger chafed at the notion of approving the Duchenne muscular dystrophy drug golodirsen on the same unvalidated surrogate marker, this time in the face of infection and renal toxicity concerns; OND’s Peter Stein granted Sarepta’s appeal while also calling attention to the company’s failure to timely conduct the eteplirsen confirmatory trial.

Rare Pediatric Disease Designation Requests Skyrocket Ahead Of Voucher Program’s Sunset

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