Austin and Max Leclaire are brothers. Austin is older, Max is younger. Like most siblings, they have many things in common and just as many that set them apart. For now, though, their strongest bond is over something they share — Duchenne muscular dystrophy (DMD), the most common lethal genetic disorder among children. Duchenne’s hallmark is increasing muscle weakness that eventually makes it hard to breathe and confines kids to wheelchairs, which is where Austin finds himself these days.
It is a genuine dilemma in this age of new-fangled "molecular" drugs, that some seem to work very well during their first human trials, but we have to wait years to get them to the public.
Of course there is massive cost to consider in some cases, but I don't know about eteplirsen. Public health advocates can have a terrible time deciding if a few people can have an expensive drug vs. a lot of people getting a cheap one. For instance, how many doses of polio vaccine would you trade for one of eteplirsen? These are the realities of government budgets. I wouldn't like to do it, would you?
