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Permalink Reply by Kenny Chu on October 21, 2014 at 9:45pm

who knows more about follistatin Gene Therapy?

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Permalink Reply by Sharon Hesterlee on October 23, 2014 at 3:23pm

Kenny, here's a link to Dr. Mendell's presentation at the conference this summer:

http://videos.parentprojectmd.org/conference14/audio/03_Saturday/S0...

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Permalink Reply by Denise Bretsik on October 30, 2014 at 7:53am

Sounds very promising. Would the trial for Dmd be a phase one or two since there was no safety issues?

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Permalink Reply by wainaina muiruri on October 31, 2014 at 10:43am

Quite interesting.No adverse effects too.

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Permalink Reply by Moein on November 4, 2014 at 12:38am

Are they going to start a new trial for DMD?

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Permalink Reply by Sharon Hesterlee on November 4, 2014 at 10:15am

The DMD trial would be an extension of the Becker trial. 

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Permalink Reply by Moein on November 4, 2014 at 3:02pm

Hi Sharon,
When would be the extension study?

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Permalink Reply by Sharon Hesterlee on November 4, 2014 at 3:51pm

Hi Moein:  I don't think Dr.  Mendell has shared the exact time line for that study publicly but my impression is that it would start recruiting within the next few months.  He will be using the same Investigational New Drug protocol that was approved for BMD so that reduces the regulatory hurdles.  At least that's the plan.

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