steroids and supplements are the only thing to do right now. Your son is young, if he were mine, I would have begun steroids as well (we did in fact when mine was 4). You have time...My son is 9...I hope i do!
Hang in there, we will all be ok!....SOMEHOW!
It's hard to have hope, but it is important to try as much as possible. The steroids are so helpful and have benefits beyond the here and now. The side effects are not as scary as I had once thought but still important to watch.
My son is 5.5 years old, not much older than your son. Connor is now grappling with the "why me?" Holding onto hope while maintaining some sense of reality has really helped us and Connor when we talk about this with him. We let him know the doctors and parents are working hard find better treatments and a cure. We are very encouraged by this and until we see the fruit of this labor, we have to deal with what we have now but always maintain hope that we'll never have to buy the wheelchair. He seems to like these explanations. He seems to hate DMD less because of it. So your hope is important to his morale too.
Hang in there. Stay realistic. Stay prepared but always hope.
Sometimes it's impossible to stay positive. Everytime I lose faith I must force myself to only seek happy things for a day, to change my mood since my sons are well worth the effort.
Then you can also focus on upcoming approaches that appear strong. Just received notice that utrophin upregulation is still on the menu, which I had given up on awhile ago. Go to www.summitplc.com to read their latest announcement. It may brighten up your day, did mine :)
My son is 20 with a large deletion as well. Hard to tell exactly if it in in-frame or out of frame. Depends upon how you interpret it. And those large deletions take awa y a big part of the protein.
john hates having DMD but focuses on what he loves to do which is art and tarantulas (the World's easiest pets!!).
Like all of us, as long as he is busy, he is happier. None of us know what lies before us. With our kids we just have more information than most. So much has changed since I adopted John at 4. Hopefully things will continue to get better. If we can transform DMD into a chronic condition rather than a "seen as death sentence", that is a s good as it gets for many conditions.
Observe and see what your kids like to do and then invest in that. Felling like you are accomplishing something.... We all need that.
Steve - I believe you are correct. Gene therapy is not mutation specific. It's basically trying to replace the dystrophin gene with a working copy. The challenge has been that because of the size of the dystophin gene, they have to use a shortened version to have it "fit" on the virus that carries it to muscle. That's how I understand it. Keith
Steve Dreher said:
Hi - You should also note the positive news that came from Summit corporation recently (SMT C-1100 - http://www.drugs.com/clinical_trials/summit-reports-progress-duchen...), it's another utrophin up-regulator. Theoretically, utrophin will help all of our sons.
I have been interested in Jeff Chamberlain's work at the U. of Washington (http://depts.washington.edu/chamblab/) - I THINK the gene therapy model is not mutation-specific.