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 »
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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 »
The Race to Yes
“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… Read more »
Top Scientists and Mothers Appeal to Congress for Breakthrough Child-Saving Therapy
WASHINGTON, Feb. 10, 2014 — Health staffers from over 80 Congressional offices together with agency and industry officials jammed a briefing room last Friday to hear mothers, clinicians and leading researchers call on the FDA to accept and promptly review a new drug application for eteplirsen, a promising new treatment for Duchenne Muscular Dystrophy that… Read more »