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Archive for April, 2014

Apr 14

A victory for the Duchenne community

Our community received the best news I’ve heard since Jack’s diagnosis two years ago.  A drug called eteplirsen, which has been the subject of many starts and stops at the FDA, has received the green light for an NDA (New Drug Application) filing by the company developing it, Sarepta Therapeutics, by the end of this year.  If all goes as planned, this NDA filing will represent the first-ever approved drug for the treatment of Duchenne muscular dystrophy.  Read the Washington Post article here.

This news comes after a huge grassroots effort by the parents of boys with Duchenne to ensure the FDA is reviewing the appropriate data behind eteplirsen–or any other safe, effective drug to treat Duchenne for that matter–and providing regulatory guidance to move therapies to market as quickly as possible for our boys.  For additional information about this campaign, visit theracetoyes.org.

Although this particular drug will only help approximately 13% of boys with Duchenne, it will pave the way for other compounds in the pipeline to move forward more quickly and into the patients that need them.  A huge thank you to all of our supporters at home, across the country and around the world that helped make enough noise to ensure we’re heard.  And to all the local news stations and newspapers who were interested enough in our story to run it and draw much needed attention to our plight.  We will not stop until our boys and ALL boys with Duchenne have a fighting chance.

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