My son aran is 8 years old and has nonsense mutation that ptc124 has the potential for a treatment.our hope is he gets the drug in the 2b trial and we pray it treats him.We are desperate for every child to be 100% treated.
Name(s) of child(ren)/individual(s) with Duchenne:
Age(s) of child(ren)/individual(s) with Duchenne:
Comment Wall (6 comments)
You need to be a member of PPMD Community to add comments!
Hi... my son, Saij is also 8, and has the same nonsense mutation/stop codon as your son. We were not able to take part in the trial, but are also optimistic about the results... do you have any latest news on the continuation of the trial? Would love to connect and exchange notes with you.
We are moving to London in the next 2 months. Who is your son's doctor? Where abouts in London are you? I am trying to get as much info as I can before we arrive. We will be living in Beckenham, Kent. Maybe we can organise a UK Families group?
Thanks for the offer--I might take you up on the it, esp. the advice on supplements. This is all so new to me and supplements in general are foreign to me. So I need all the help I can get in that area (as well as many others!). Take care, Jill
I do hope all goes well with PTC124--every day and every minute counts with our boys, so I do pray that your son will start receiving the medicine soon and that it will do what it was created to do! Exciting times in the DMD world. Keep me posted! Cheers, Jill
Just wanted to drop you a line and say hello. What part of London are you in? We lived in Woking for a couple of years and loved it and the culture there. (And we loved all the travel living there afforded us! So much to see and do.) My husband is acutally flying home from there tomorrow. He said the weather was fabulous this past week--he so wishes we were still there. We live in hot, humid Houston, Texas! We love it here but it is a little too hot in the summer months!