Our Update to the Community

Posted by: Christine McSherry on February 09, 2014


You helped, and we owe you an update…so here it is!!  Thanks to your outreach efforts, Friday’s congressional briefing was packed.  More than 100 people jammed the room to hear about Duchenne and the urgent need to use accelerated pathways for approving new treatments.

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Click here to watch a 5-minute highlights video.

What’s next?  We are following up with all of the congressional offices who attended.  Our “ask” is that FDA accept and promptly review a New Drug Application for Eteplirsen, a promising new treatment that can help 13% of people with Duchenne.  The quicker this drug moves through the regulatory process, the quicker similar drugs will be developed.  One of those compounds waiting on the shelf can help Charley.  We need to get Eteplirsen approved so the children who can benefit from this first drug get treated without further delay.  And so the follow-on drugs can move forward.  Not years down the road.  This year, now, 2014.

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