“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 »
If you’re visiting the Capitol Complex today, you’re likely to meet a band of sisters – six moms of children with Duchenne. Quite literally the team is “Running the Race to Yes” in an effort to visit the offices of 100 U.S. Senators and each of the 435 Members of Congress carrying an urgent message… Read more »
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 »