Regulators block Duchenne muscular dystrophy treatment after fatal side-effects outweigh questionable efficacy

In its own communique issued shortly after the FDA’s, Sarepta elaborated on the situation and confirmed a Roche statement to Bloomberg News that linked the probe to a fatality in Brazil and held that the death of the patient was unrelated to Elevidys itself.

In a major win for Sarepta Therapeutics Inc., US regulators are recommending that patients who can walk be allowed to take its gene therapy Elevidys again.