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Sarepta Therapeutics said Monday that a second-generation medicine for patients with a certain type of Duchenne muscular dystrophy showed improved performance in laboratory tests over the biotech’s marketed treatment.

In a small clinical trial, a monthly infusion of the new Sarepta drug, called SRP-5051, produced eight times more of the muscle protein dystrophin compared to weekly infusions of Exondys 51, the company’s existing product.


Signs of kidney toxicity — a particular area of concern for SRP-5051 — were not observed in any of the treated patients. However, two patients reported low levels of magnesium in their bloodstream that were considered severe adverse events.

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