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

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.

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.