Sarepta Provides Update on Clinical Trials Planned for 2014

Streamlining exon skipping drugs for other mutations will allow the company to reach over half of the Duchenne muscular dystrophy population.

On May 13, Sarepta Therapeutics provided an update on the clinical trials they are planning to begin later this year for specific subpopulation of boys with Duchenne muscular dystrophy.

The team at Sarepta are juggling with a lot of variables as they move forward with their clinical trials. They need to plan for a pivotal clinical trial that will convince the FDA their drug is safe and effective. They need to continue with the trials currently underway so that those boys can continue to thrive and provide additional data for the FDA.

They need to develop their clinical trials in the hope that they will get accelerated approval which in turn will provide a template for similar drugs they have in development. And they have to do it all quickly because investors and patients are desperate for results.

With that in mind, the team at Sarepta held two webinars [May 13] — one for investors and one for patient families.

In the webinar intended for the families of Duchenne muscular dystrophy patients, executives from Sarepta provided an overview of the clinical trials they have planned for 2014 and how families can get prepared to see if they are eligible to enter the trials. In his introductory remarks CEO and President of Sarepta, Chris Garabedian reminded the audience that Sarepta received guidance from the Food and Drug Administration (FDA) recently to assist the company in developing their clinical studies with eteplirsen for regulatory approval.

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