There is undoubtedly greater hope than ever before in the world of DMD. More funding is going into research than every before, and yet, with all of the research that is going on, clinical trials have yet to demonstrate convincing efficacy to the necessary regulatory agencies. Unfortunately, the plural of anecdote is not data, but stories, like that of Billy Ellsworth, suggest that the drugs work, and a cure is right in front of us.
Today, Biomarin, the producer of drisapersen, is going before the FDA to seek approval of their drug to be distributed to individuals with the appropriate genetic mutation.
Just recently, the Washington post eloquently highlighted the hope that our DMD family shares as we continue to fight this disease. The article (and associated video) are certainly worth a quick glance.
I'm a current MD-PhD candidate, working hard to help treat and manage muscular dystrophies