Sarepta and Prosensa Announcements This Week

There continues to be good news for the Duchenne community.  We will continue to monitor Duchenne research news and share it with you as it becomes available. Here are three announcements from this week:

Sarepta Therapeutics Announces Publication of Eteplirsen Clinical Study Results in the Annals of Neurology

Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced the first peer-reviewed publication of the 48-week results from the Phase IIb clinical study of eteplirsen in the Annals of Neurology. Eteplirsen is an investigational medicine in development for the treatment of patients with Duchenne muscular dystrophy (DMD) who have a genotype amenable to skipping of exon 51.

Published study results showed that once-weekly treatment with eteplirsen resulted in a statistically significant increase from baseline in novel dystrophin, the protein that is lacking in patients with DMD. In addition, eteplirsen-treated patients evaluable on the 6-minute walk test (6MWT) demonstrated stabilization in walking ability compared to a placebo/delayed-treatment cohort. Eteplirsen was well tolerated in the study with no clinically significant treatment-related adverse events. These data will form the basis of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for eteplirsen planned for the first half of 2014.

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Sarepta Therapeutics Announces Second Quarter 2013 Financial Results and Recent Corporate Developments

Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today reported financial results for the three months and six months ended June 30, 2013, and provided an update of recent corporate developments.

“We are excited that the FDA is open to an NDA filing for our drug eteplirsen for the treatment of Duchenne muscular dystrophy and the organization is focused on all of the activities necessary for a successful NDA submission in the first half of next year,” said Chris Garabedian, president and chief executive officer of Sarepta Therapeutics. “The Sarepta team is planning for success as we continue activities related to our eteplirsen confirmatory study and our manufacturing scale up, while we begin pre-commercial activities to prepare for the potential approval of eteplirsen.”

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Prosensa to Present at Wedbush 2013 Life Sciences Management Access Conference

Prosensa Holding N.V. (RNA) announced on August 6 that Hans GCP Schikan, Chief Executive Officer, will present a corporate overview at the Wedbush 2013 Life Sciences Management Access Conference in New York, NY on Tuesday, August 13, 2013 at 1:55pm ET. A live webcast of the presentation can be accessed through the Investor section of the Prosensa corporate website at and will be archived for 90 days.

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