Monthly Archives: April 2014

Paying the Price

by Marissa Penrod Yesterday was a historic day in the world of Duchenne. Sarepta Therapeutics announced that they’d finally received guidance from the FDA on moving the first Duchenne treatment through the approval process in an expedited manner. It may be coincidence, but it’s beautifully symbolic that, on the morning of Patriot’s Day, this news… Read more »

Thanks to YOU, a Fighting Chance…

With the announcement today, our children facing Duchenne now have a fighting chance! Thanks to each of YOU who signed the White House petition, and cheered The Race to Yes, from your neighborhood to the halls of Congress and the FDA, we did it. To celebrate this momentous occasion, please join us for a national telephone… Read more »

5 Reasons WHY You Should Join Us In This Race!

1. Because Duchenne is the leading genetic killer of children – it has a 100 percent fatality rate. 2. Because there is a drug that has been proven to be safe and effective, eteplirsen, that could potentially save thousands of lives, but it needs FDA approval. 3. Because over 100,000 people spoke and petitioned the… Read more »

Parents Ask FDA to Approve Duchenne Muscular Dystrophy Drug. Race to Yes Seeks Decisive Eteplirsen Decision Within 30 Days

See the release online WASHINGTON, April 9, 2014 — Parents of children facing death from Duchenne muscular dystrophy today asked the U.S. Food and Drug Administration to approve in 30 days a well-tested drug that halts the disease with no side effects. The parents, representing The Race to Yes campaign, challenged FDA Commissioner Margaret Hamburg to take the steps needed… Read more »

This Plane Has Been Cleared For Takeoff

It’s old news by now…..Sarepta’s exon-skipping drug, Eteplirsen, is safe and shows efficacy. If we sift through all the details, all the facts, and all the distractions, we’re always left with the same basic, essential information – Eteplirsen is an appropriate candidate for Accelerated Approval.  Duchenne is severe, rare and has no therapy.  Duchenne is… Read more »