First, your son's mutation may well fit into the exon 51 skipping strategy, though mutations are a bit more complicated than 48-50. Information about specific exact breaking points must be understood but as a general statement, your son fits into this strategy. Several companies (prosensa and avi biopharm) are involved in trials targeting exon 51. At the moment the trials are concerned with safety and dose escalation, in order to figure out how much is needed in order to achieve sufficient dystrophin expression. Your last question about excitement - yes, this is a very promising strategy. Trials will be expanded into the US in 2009. I am not certain about Canada but clearly in order to recruit sufficient numbers of boys into these trials, there will need to be a number of clinical sites involved. Your question about 'is this the answer we are paying for" - this strategy attempts to change the duchenne phenotype to a becker, a slower progression. We are all hopeful exon skipping is a major step forward. The goal is to get proof of concept from these early trials. Keep praying.
Someone asked here about deletion 50,51 and Linden detabase . (2 patients diagnosed withDMD). My son can be example of debate if he is DMD or BMD. From genetic mutation he is DMD . By the symptoms ,I was already told by few doctors and other people in the field that he looks like he is Becker. Personally for me is not important how they will name it. They can call it BMD or Mild DMD, What is more important for us , what they can do about it . Clock is ticking.I already do not expeckt any big miracles with my son being 9,5 years old and exon 51 just starting trials , when 53 is what we need. The only miracles I can expect is the miracle in known pharmacology or stem cells.In mean time I do many things in my life to make sure that I do not hear the clock. I fixed my car today for example.. BUT no matter how busy I stay , how loud I am , I hear the clock .Good luck.As younger your kids are ,the biggest chance they have .