i think it would help me be able to do be able to do the things i like and for it to be easier for me to do them, like play the drums. joining me now from washington is jen mcnary. you met her in the piece, austin and max s mother. the fate of this drug has been in limbo for quite some time. the fda did lay out a road map for the company, to conduct more trials, to get the drug possibly to more children and potentially lead to its approval if the ruls pan out. jen, you and i had a long conversation about this. the new trial is not going to launch for a few months. when it does, first thing is first, will austin be able to get the drug, do you think? you know, the fda has allowed for a flexible trial design with no placebo, including nonambulant children. so, in theory, austin fits. again, we don t get special treatment. he will be in line with all the other boys that could possibly benefit from this drug.
and jen believes it s due to an experimental drug. max began weekly infusion treatments of the drug 2 1/2 years ago, part of a small clinical trial of 12 boys. austin didn t qualify because he was already in a wheelchair. how much of a difference has it had in their lives? i think you re literally looking right now at the mother of two children with duchenne, one who is going to be the first child to survive the disease and one who is going to have the last child to die from it. since the trial started all 12 boys have seen their symptoms start to stabilize and don t sooeb seem to be many side effects. the fda still has not approved the drug. they want more evidence. the fda says look, we re just not sure yet. this is part of why we exist, to be sure that it is safe. yes, it s been a two-year trial. but all these kids give them more time. we re not sure how effective it is because, again, it s too
small. we have said that we will accept the possible risk that this drug isn t as effective as we think it is five years down the line or some kind of side effect develops. but in the meantime, while they are not sure and while they are collecting more data, our kids are dying. frustrated with the process, parents like jen have become vocal advocates, pushing for this drug s approval. it s become a contest of the emotional stories of these children and parents versus the very drug approval process itself. i want to know that they understand what children go through in a clinical trial. finding therapies for rare or orphan diseases like duchenne can be difficult because of the small number of patients and the extreme progression of the disease. by definition, rare diseases have fewer than 2,000 cases in the country. in the case of duchenne, there are just 15,000. rare disease is such that if we don t get our children in clinical trials and get these drug out quickly, there s n
15-year-old austin mcclaire has always wanted to be part of the high school band but day-to-day it s becoming harder to play. austin s arm function is getting worse. i can see him struggling to lift his drum sticks. austin has a severe form of muscular dystrophy. it s the disease that jerry lewis has been raising money for, for the past 60 years. have you noticed anything over the last couple of years that s become more challenging? it s been harder to get in my bed and position myself. having one son with this disease would be hard enough for any mom. jen has two. austin and his younger brother, max. boys first lose the ability to walk around the age of 10. most don t live past their 20s. his brother defies these odds,