Is there anyone who has gone through the trial screening or even potentially getting the drug in the 2b trial,if so how long have you been in the trial and please share your thoughts.It would be helpful for us and many more who are waiting to get in the trial.
We tried to get on the trial, but James has just turned 4 (in May) so they considered him too young. We actually fought hard to get him included, but they were very strict about their criteria. Hopefully, they still won't have the numbers by the time he is 5 and we may get him on the trial early next year, as I hear they are still trying to recruit boys.
I would also be interested to hear from anyone whose son participated in the last trial.
My son participated in the 2a trial 2 1/2 years ago. Jack had his biopsy, etc. last week for the 2a extesion and will go back to Utah next week to start the drug. If you have questions that I can help with, please contact me directly.
I am not sure where you got that from about non ambulant kids participating in the ptc trial,I know ptc is very tough on the set criteria and only currently taking ambulant kids.My hope is the drug works and benefit the boys with nonsense mutation and other drugs come out soon to benefit everyone.Although i can do with everyone involved in running the trial to not take so long to start everything.We are in UK and trial centre we will go to has not even started recruiting and we have been waiting since march this year.
How old is your son and does he have point mutation.
The confusion might stem from the two trials that are happening simultaneously. The 2b trial does require boys to be ambulatory but the 2a extension trial requires that the boys participated in the original 2a trial. At the time of the origianl trial all boys had to be ambulatory but to enter the extension 2a they may no longer be walking and are still eligible because they were in the first trial. I hope this helps. My son Tim was in the first trial and we are about to start the screening process for the 2a extension in Cinn. We saw great benefit the first time and are very hopeful for this new trial. The blood work and biopsies were difficult but Tim made it through and is ready to do it again.
Our son Jacob (age 9) started the 2b trial in Philadelphia 3 weeks ago - that is when he first got the drug/placebo. It was a little difficult getting used to the dosing three times a day as prior to this he only took meds in the morning. We are in a routine now and everything is going well. We hired a sitter to come into our house to watch our preschooler and so that Jacob could come home after school. He takes the midday dose when he gets home from school - which is why I wanted the sitter. This way I don't have to count on the school to do the dosing and return the empty packets. It makes it easier on Jacob too because he doesn't have to be singled out to take medication at school. We have seen no change yet: but today he seemed like he was going to jump out of his skin he was so hyper - don't know why that was. He had bloodwork done this morning and he gets upset when that happens so that could be why he was so antsy. We'll have to see how he is tonight.