Dears, I am a father of fantastic young boy, 8th years old. As a child who are just entering his life he has many dreams and plans: who to be in a future, what to do in the future... However, the future may not be for him, at least this future he things about. He is DMD affected. He is already excluded from the activities that his friends do (e.g. futball, othe sports...). He feels worse than others. Decrease of his strength and increase of his awareness make him finding it as not a temporary condition, but as really the illness; which progresses.
Recently we told him that scientists work on the treatment development and there might be a day that the drug will be available and bring a cure. He answered - "Nothing appeared for many years, I do not believe it will happen in one next year". Crying!!! This one year is whole life for him.... And in parallel only a bit of time in the drug development and approval process. (example - year ago application for conditional approval of Ataluren has been accepted by EMA; we may rather say - it has not been rejected; no response so far!). One year is a huge time for our boys, for their life perspectives. In parallel with physical condition, the psychological one weakens. His and ours, whole family. Life is harder and harder. Everyone around say - you have to manage that, you have to take care. We do. We try. But should we? Every day? How? Where to draw the strength from? We are exhausted. The DMD itself plus steroids devastate our son's organisms any way. Their life horizon is inevitably closer, with no hope. Could conditionally approved Ataluren or Eteplirsen be really worse scenario??? How do you think? Could our boys get a bit of hope? Does not they deserve that? Their clocks are ticking. Support them please!!! Best Regards, Grzegorz