Hi Linda:
Thanks for your comments. My heart goes out to you with two boys going through the same. Yes, this disease is so unfair and the worst of it is that we are totally helpless and cannot "fix" it. Liam's got exon 48, 49 & 50 missing. At this point, we don't know if it's Becker or Duchenne. I lean towards Becker because of the fact that Liam is nearly 9 with very few symptoms other that being slower than his piers when running. Liam does not present with Duchenne, but until a muscle biopsy is done, we really won't know for sure. I still look back with a very critical eye and see no signs that would lead me to even think of DMD - even knowing what I do now, I would not have caught anything. Liam even went to a ped. neurologist last summer who never thought to even run blood tests. My fear is that our current doctor, RObert Leshner, at Children's in DC, has just lumped Liam in with all his patients. Yes, the decisions are left up to us as parents - even the brunt of the research must be done by the parents and put forth to the doctors. And, I fully agree with you on the quality of life issue. For Liam, to be confined to a bed and be kept alive with tubes and wires is no quality of life at all, but if there is something worth fighting for, then we do. I was very hesitant to put Liam on Prednisone because of the possible behavioral changes that can occur in some boys. If Liam does not remain the Liam we know and the drug changes his personality so much, I will stop it in a heartbeat. But for this first month of it, he seems to be tolerating it very well and is still the same kid, just a wee bit more excited. I see NO other changes in strength - absolutely none. Last summer, Liam could not do a sit up, although he ran like crazy, just slowly. Nor could he jump with two feet more than a few inches. Now Liam does sit ups and jumps more than 20 times in a row. All this was done with out steriods - pre steriods if you will.
Liam has gotten stronger during the past year - before we even realized this was MD. That is what I certainly do not understand. Liam is strong, goes up and down stairs without a care in the world. He runs, jumps, rides horses, swims, creates obstacle courses in the back yard to do and rides a bike with training wheels. I just don't get it. We have thought many times about switching the the other steriod and will probably do so at the end of this month because it seems better for the heart in the long run and fewer side effects than Prednisone. If we do switch and notice anything negative or adverse, we will switch back to Prednisone. I feel confident, at this point, Liam can benefit from these drugs, but, like you, if I see no reason, they will be stopped right away. I have also done tons of research on other therapies including green tea, curcumin and others supplements. We will try anything that doesn't cause adverse side effects - anything that is evidence-based. Liam, with regular exercise, OT and PT - along with riding and swimming, is doing fine at this point and I just have to think about these drugs because I do not see the physical strength improvement - they may be helping in ways that are not apparent on the outside. I just don't know.
You are doing what's right for you and your children and in reality, quality of life is the ultimate in importance. I admire your strength in so many ways. I did just read about the twice weekly drugs in higher doses instead of the lower daily dose. Did you find this to be beneficial for you boys?
Well, your boys are lovely and give then perfecto hugs from us all here in VA.