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Sarepta Marks A 'Very Important Day' For The DMD Community

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Sarepta Marks A 'Very Important Day' For The DMD Community
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Shares of Sarepta Therapeutics Inc (NASDAQ: SRPT) soared by more than 10 percent Wednesday after the company reported encouraging results from a clinical trial of a therapy intended to treat Duchenne muscular dystrophy (DMD).

Sarepta's drug called golodirsen showed that patients who suffer from DMD and took the therapy saw an increased production of the protein dystrophin to 1.02 percent of normal levels from 0.095 percent without the drug.

"Today is a very important day for the Duchenne muscular dystrophy community," Sarepta CEO Doug Ingram exclaimed during CNBC's "Squawk on the Box" segment.

Sarepta's golodirsen is the company's second drug for the treatment of DND, the first being Exondys 51 which was approved last year. Exondys 51 can only treat around 13 percent of patients suffering from DMD and the company's goal is to treat 100 percent.

"The data we had this morning shows we are on the right path and we took a significant step forward," Ingram said.

Looking forward, it's too premature for Sarepta to apply for an accelerated approval for golodirsen, Ingram said. The company has several other ongoing programs and trials and the more appropriate step would be to open a dialogue with the U.S. Food and Drug Administration as to what it will take to bring the therapy to patients who need it.

Nevertheless, the data read-out was "extraordinarily positive" and validates the company's approach it's taking to treating DMD and hopefully a step forward to treat everyone who is suffering from the disease, Ingram concluded.

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Posted-In: DMD Doug Ingram Duchenne Muscular Dystrophy Exondys 51CNBC Health Care Media General Best of Benzinga

 

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