Monthly Archives: March 2014

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 »

You Can Bring it to a Boil

At 211 degrees water is just really hot. But one more degree makes it boil. One degree shifts it from a really hot liquid to a force that can power locomotives. One degree. One that matters. In 1920, Tennessee was the final state needed to ratify the 19th amendment that allowed women to vote in… Read more »

Never, Never, Never Give Up

“Never, never, never give up!” Winston Churchill once said this. When asked what her favorite motivational quote was, Olympic Gold Medalist Aly Raisman said that this quote was what inspired her – it’s what motivates her to never give up. So what do an Olympic Gold Medalist, Winston Churchill and The Race to Yes have… Read more »