Is anyone currently enrolled in the phase II or phase IIa PTC 124 trial? We are currently finishing the pre-approval process with getting a biopsy next week and then onto the medicine Feb. 1. Looking…Continue
I remember when my friend Sheri called me to tell me her son was asked to be in this study, I think I cried all day for them. The DMD community is such an amazing group of people and I pray that there can be a cure for all but for now I pray the trial is a huge sucess! It would be such amazing progress in the treatment of this disease. When TYler was diagnosed steroids were the biggest thing. There is so much hope on the horizon! Please let me know when Brandon starts the trial. I know Troy strarted it last week. I will let you know if I hear anything.
No Tyler is not in the trial. He has a duplication so we are hoping and praying for Exon Skipping to hurry along. Our friend Troy is starting it. Today was the biopsy and he is suppposed to start the drug next Tuesday. Are you going to be able to attend the DMD trivia night 2/20/10? Kids are invited and welcome. We'll be there. Hopefully you can make it. Your photographs are beautiful!!!!
You ask why would we friend someone we have never met? Because we are all in the same boat and because I was simply drawn in by the great profile picture! Totally awesome! I don't have any information on the PTC 124 trials, but wanted to come over and tell you I loved your picture. I am Jennifer, mom to Xavier, age 8 with DMD deletion 48-52. Our family lives in Shakopee, MN. Nice to Meet you!