European regulators on Friday said that a Duchenne muscular dystrophy gene therapy should not be approved, further imperiling the future of the treatment that is facing increasing scrutiny in the U.S. and globally.
The decision in Europe comes as Japan and Brazil paused any distribution of the therapy, called Elevidys, while safety concerns are being investigated, meaning it is not being used anywhere in the world for now. Brazilian authorities also disclosed that a boy there who received Elevidys later died, though his doctor said the cause was not related to the therapy.
Elevidys was developed by Sarepta Therapeutics and is marketed outside the U.S. by Roche.
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