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Sarepta Higher After Progress With Muscular Dystrophy Drug Trial

Sarepta's trial supports its drug's 'potentially differentiated profile' to treat Duchenne muscular dystrophy, the company said. The stock is higher.
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Sarepta Therapeutics  (SRPT) - Get Sarepta Therapeutics Inc. Report shares jumped Tuesday after the drugmaker reported progress in a trial of its Duchenne muscular dystrophy treatment.

The trial was conducted with Roche RHHBY.

The Cambridge, Mass., company's shares recently traded at $84.12, up 12%. They have slumped 40% over the past six months.

“In results from the first clinical study using commercially representative material, SRP-9001 demonstrated robust expression of micro-dystrophin and no new safety signals from prior studies,” Sarepta said.

That “supports [the drug’s] potentially differentiated profile for the treatment of Duchenne muscular dystrophy,” the company said.

“SRP-9001 is an investigational gene transfer therapy intended to deliver its micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein.”

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In the study, 20 participants ages 4 through 7 were treated with a single infusion of SRP-9001. Symptoms of DMD usually appear in infants and toddlers.

In muscle biopsies from the first 11 patients taken 12 weeks after treatment, “all patients demonstrated robust transduction, with mean micro-dystrophin expression of 55.4% of normal, as measured by western blot,” Sarepta said.

Duchenne muscular dystrophy is a fatal neuromuscular genetic disease that afflicts 1 of every 3,500 to 5,000 males worldwide. 

DMD is caused by a change or mutation in the gene that encodes instructions for dystrophin. This is a protein that is required for muscles to work properly and is missing or lacking in people with Duchenne. 

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