What drew me to submit my story is that PPMD stated they are working for all potential treatments for DMD to be fast tracked thru the FDA and not just one specific treatment. As the grandmom of a 12 year old with DMD it's very hard to sit back and watch Jacob get weaker and weaker and knowing that he will soon be in a wheelchair and worrying how will I ever handle him by myself to have him over like I do every weekend now is just overwhelming! I can give advice about treatments and care that I've read about from others to my daughter and son-in-law but in the end as frustrating as it is I have to honor what they decide. I know that the same goes for the FDA's decision but it too is very frustrating. I often feel so out of the loop with everything being so secretive and only a select few having one on one meetings with people from the FDA. All we ever hear is that we can't talk about specifics! I have to believe that by now those responsible for deciding the fate of clinical trials know all about DMD and the effects on the children and the families so I'm putting my faith and trust in you to do the right thing.
Please if these treatments show any promise of helping, with a minimum of side effects I ask that you give these children a chance! I've heard people say that they worry what might happen down the road with these drugs as far as side effects. That's all said and good but we know for a fact what will happen if we don't try. We all know that time is of the essence and we all know that the boys already non-ambulatory need these drugs even more that the ones that are still ambulatory. There needs to be a set of standards for trials for these boys decided on now, not down the road. As someone on the sidelines I don't understand why meetings have to be set so far apart, why so much time seems to be wasted and why these standards can't be agreed upon now! It would be nice if the FDA could set up a page for the parents to go to express their thoughts and concerns and to be up on the latest news about these trials and decisions that are being made.
My hope is that the FDA and pharmaceuticals companies will come to agreements as quickly as possible and all of this generation of boys will at least have some sort of treatment besides prednisone! Sincerly, Jacobs Mommom