Say Yes with Us!

Posted by: Christine McSherry on March 24, 2014


It’s time for a show of hands.  Who stands with us in the fight to approve eteplirsen and make this the first generation of boys to survive Duchenne?

As of this morning, more than 82,000 people have added their names toTheRaceToYes.org petition that will involve the White House in our fight to gain FDA approval for this miracle drug.  There’s only one word for this level of support: AWESOME!

But do the math: we’re still 18,000 signatures away from our goal.  If you are reading this, PLEASE push your friends, family or colleagues to sign the petition at TheRaceToYes.org.  We’re posting a new video about our petition, and it’s a great way to tell people the story about what we are doing.  (Duchenne may be a genetic disease, but we’re hoping the video goes VIRAL!)

The Race to Yes Video

Why are we doing this? In other words, why do we even find ourselves in this position of fighting the FDA about a drug with great promise and zero side effects?  I think it’s worth reviewing what brought us to this point, and our story can be summarized in five points:

•    Duchenne is a death sentence – period.  There has never been a treatment to halt the progression of the disease. Without a treatment, boys with Duchenne stop walking at 10 or 11, and most will not see their 20th birthday.

•      All of that changed when a new drug – eteplirsen – announced 48 week data proving that boys with Duchenne stabilized after treatment. Then…one year passed, and we saw 120-week data that confirmed that some stabilized boys even IMPROVED their six-minute walk distances!

•    Everyone in the Duchenne community held their breath, and waited for the FDA approval that we just KNEW was around the corner.  And of course, we’re still waiting. EVERY DAY OF DELAY means that EVERY boy with Duchenne slips further in the grip of the disease.  (The exceptions, of course, are the 12 boys on eteplirsen. That includes twins who continue on eteplirsen and prove that even boys who cannot walk can benefit from the improved pulmonary function that eteplirsen provides. This drug clearly is a miracle.)

•    So…WHY do we continue to wait for the FDA to grant accelerated approval? We don’t know. Are there people at the FDA who fail to recognize that Duchenne boys have EVERYTHING TO GAIN and NOTHING TO LOSE by taking eteplirsen??

•     By signing TheRaceToYes.org petition, 100,000 people will involve the White House in our fight. If the FDA won’t listen to boys and families struggling with Duchenne, perhaps they will listen to the President of the United States when he asks a simple question: Why have you waited nearly 18 months to give boys with Duchenne a chance with a drug with ZERO SAFETY ISSUES?  The proof is in the data – and I like to quote the Australian researcher who spoke at our congressional briefing in March: “It’s bloody obvious” that eteplirsen works!

OK, that’s enough from me.  It’s time for everyone to scroll through their phones and find one, five or 10 contacts who can be encouraged to add their names at TheRaceToYes.org. Tell them to watch the video, and add their names to the list of people who are determined to save the lives of thousands of young boys with Duchenne.

We are so so so so close.  Let’s make this happen!!

 

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