“Lucky” isn’t a word that usually comes to mind when thinking about children with Duchenne. The disease devastates kids and their families. It is 100% fatal. But, in a way, for the first time, we are fortunate. We have known what causes Duchenne for over 20 years. Affected children do not produce a crucial protein called Dystrophin. As a result, their muscles break down and they lose mobility and function and, ultimately, their lives. Great minds in science have devoted their lives to cracking the code on Duchenne. For decades, there have been necessary failures, massive community advocacy, grassroots and government funding, and numerous clinical trials.
It has been a long and grueling marathon. But now, with a real potential treatment waiting for FDA approval, it has become a sprint. A race to get this and future therapies to patients. A Race to Yes.
Eteplirsen is the first Duchenne treatment with robust clinical data showing safety and efficacy, and consequently, we are faced with the miraculous yet sobering reality that this generation of Duchenne children could truly be the last to die or the first to survive Duchenne. We are that close. Our children deserve action. Days count. Hours count. Minutes count. We have a real chance to cross the finish line. It’s time.
The month of February has been a remarkable one for Duchenne advocacy. Several weeks ago, over 100 Congressional aides attended a briefing to learn about the urgency to approve Eteplirsen. They heard from three of the world’s top Duchenne experts who agreed that this drug deserves accelerated approval. “This drug works,” one expert stated. “It’s bleeding obvious,” added another. Additionally, Congress again heard from our community this week about the critical importance of re-authorizing the MD Care Act.
We are fortunate to have champions in Congress. Senator Roger Wicker is leading the charge on re-authorizing the MD Care Act. Senator Ed Markey initiated a bipartisan Senate and House letter to the FDA urging the agency to make use of the law Congress passed (FDASIA) in 2012 that allows them to accelerate treatment approvals for diseases like Duchenne.
The momentum is real and the opportunity to save lives is within reach. So, today, in honor of Rare Disease Day, we are launching “The Race to Yes” on the web, on FB and on Instagram. This is our site, our place to unify efforts for one singular goal. This is our chance – to participate, to make an impact, to stay the course on The Race to Yes.