Dear Connie, Thanks so very much for your kind words. I sometimes think this is more difficult for grandparents with your heart breaking two times over - for your children and your grandchildren.
I am happy Elliott is in the PTC124 trial. I know you are anxious to see change but it is really important to remember that Elliott is in the Phase 2b trial. In this trial, Elliott may be on one of two doses (low/high) or placebo. Connie, we are all praying that ptc124 is the first treatment for boys with premature stop codons. Please stay in touch and let me know how he is doing. Participating in trials is really wonderful and difficult all at once, as we all wait and watch.