"My concern now is that the EMA will follow the CHMP's decision, and not give approval for the boys not walking. Then we have to wait for 3 more years. Our boys will be weaker, and perhaps for some - too late. We have to have the parents…"
I think this is good news too, BUT once again the non ambolatory boys are forgotten ! The CHMP's members will recommend that EMA (european medicines agency) will give approval to boys from the age of 5 and AMBULATORY.
"Thank you Sharon. Please keep us posted - good or bad news. Would also like to hear what PTC says, when you have spoken to them about the time line.
Needless to say, all boys with a stop codon not included in one of the previous Ataluren…"
Although we live in different contries around the world, I don't think there are differences in how parents relate to the risk/benefit issue. We all have one thing in common, not matter where in the world you live. We want the…"
Thank you for your reply. This paper you are working on, will it adress Ataluren specific or trials in genereal ? You mentioned earlier that having the parents voice up front when it comes to what we are willing to risk contra benefit…"
"Hello Berit. Thank you very much for your invitation. I hope we can do something to wake up those involved in the approval process, but they all seem to be entangled in a very bureaucratic approach, and they fail to see what is obvious: that the…"
"Hi Bernardo, and one more question to Sharon
Share you fear for the timeline, Bernardo. Not sure how to lobby the EMA, but Sharon mentioned earlier that having the parents voice up front when it comes to what we are willing to risk contra benefit…"
Hello Berit. Thank you very much for your invitation. I hope we can do something to wake up those involved in the approval process, but they all seem to be entangled in a very bureaucratic approach, and they fail to see what is obvious: that the Phase IIb trial was a success and that it shouldn't have been terminted. I am sorry about your personal case, with all the preparations for the trial and the sudden cancellation, etc. Well, let's keep insisting, and see if we can do something. Best wishes. Bernardo
We are actually Australian, but just recently moved to London (after 8 years in SIngapore and Tokyo). My husband has organised a bike ride from the Neuromuscular INstitute in Sydney to the Neuromuscular institute in Melbourne and will in fact have the head professor from the melbourne institute and another guy from the Sydney institute accompanying him. I am also in contact with the Sydney doctors coordinating the trial, so I might send off an email to them tongiht and ask. I'll let you know what they say. wouldn't it be great if they were still recruiting????
Quick question, are they still recruiting in Australia? I know it's a long way but it could be an option. I know the UK trial is full and so we are going to see if Australia is full and try to get James on it when he turns 5 in May. We'll do anything for him, just like you would. It was just a thought....
I have only today read your discussion on trying to get your son into the PTC124 trial. I was wondering if you ended up getting him on it? Our son is 4.5 and has a nonsense mutation, but of course he was too young for the trial. I am so sorry Magnus didn't get on the trial and I am hoping you got him on somewhere, as i somewhat understand the sadness and frustration of not being included.
The list I posted has other sites in Europe also. If I were you I would start there. Jack has been part of the 2a extention for 3 months and the travel has been much harder on him than expected. We only fly 1 1/2 hours to Utah for treatment. Though I believe the 2b is less demanding, it is still a serious consideration. Best of luck to you and Magnus, I hope you are able to get him in the trial.