Last week I was one of ~50 chapter board members to attend the Patient Focused Drug Development meeting in Hyattsville, MD. Members of the SMA community spoke before members of the FDA about their journeys with SMA, or that of their child(ren). I have an update on that, as well as a short recap of a presentation given by Avexis and why adding SMA to the list of conditions for which newborns are screened is so important. Also, a world without typical SMA type 2's and a life of powered wheelchairs and breathing equipment? Yep, I think the time is near. Links mentioned in this episode: PFDD 4/18 Video Clips Avery's Bucket List and my blog post about it from 5/2012. Most recent study results from Avexis and their gene therapy clinical trials Cure SMA's Newborn Screening Coalition Cure SMA legislative advocacy priorities MDA's statement on adding SMA to newborn screening panels nationwide