Blog

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 »

What is the holdup?

There was a key hearing with FDA Commissioner Hamburg on Thursday, March 27th, before the U.S. Appropriations Committee  – the Committee that funds the FDA. Click here to watch the full hearing. During the hearing, Congressman Sanford Bishop (D-GA)  asked FDA Commissioner Margaret Hamburg: “In the weeks worth of data found by researchers and scientists… Read more »

“The Race to Yes” Hits Major Milestone with 100,000 Petition Signers Urges President to Support Safe and Effective Treatment for Duchenne Muscular Dystrophy

Washington, DC – The Race to Yes, a national campaign by families with children facing death from Duchenne Muscular Dystrophy achieved an important milestone just after 8pm EDT last night when the 100,000th person signed their White House petition urging the U.S. Food and Drug Administration to approve a breakthrough drug that has been shown… Read more »

What’s in a Number?

Just 30 days ago, a small army of Duchenne parents and organizations launched a petition asking the White House to urge the FDA to use accelerated approval to get treatments to Duchenne patients quickly.  I’m not sure we understood the monumental task in gathering 100,000 signatures to guarantee a White House response.  One hundred thousand… Read more »

What Next?

As we approach the White House petition deadline  on Friday of this week, we are hyper-focused on two things: 1) reaching the 100k goal 2) planning what happens next First, a word on #1. The word on #1 is “wow.”  Wow describes the emergence of families who – some for the first time since their son’s diagnosis — feel… Read more »