Today (March 3, 2010) PTC Thearapeutics and Genzyme sent out a press release regarding the results of the Phase 2b ataluren trial. We know that there are many questions surrounding the information presented and we are working very hard to get the answers you need and deserve.
PPMD has been in contact with PTC and Genzyme this morning and tried to ask questions that we know are on your mind. Unfortunately, we were not given very specific answers except that the trial failed to meet primary and secondary endpoints. It was the suggestion of PTC that all patients currently involved in the trials contact their clinicians for specific information regarding their participation. While we know this isn’t the update you wanted to hear, it is all the information we have at this moment and so we wanted to make sure to pass it on to you.
In the meantime, PPMD will continue to seek out answers, ask questions, and advocate for the entire Duchenne community. We will also make sure to inform you of any updates as we get them. On Friday, March 5th at 12 noon Eastern, myself and Senior Director of Research and Advocacy Sharon Hesterlee will host a conference call for parents where you are invited to ask questions you may have and participate in discussions regarding the future of the trials. We have invited leadership from PTC to participate on this call as well. We will also share any additional information we have
received at that time. Details on how you can dial in to this conference call will be posted on our website before the end of the day tomorrow.
As always, PPMD remains committed to aggressively seeking answers for our boys. Please continue to check our website, community site, and Facebook page for up-to-date information on how you can join these discussions.
Founding President and CEO
OK, this is yet another blip since we've been aware of this drugs development in 2002. At 16 and a half our son is likely one of the oldest ambulatory Duchenne kids in the trial. Our son has had 4 biopsies within these earlier and later trials for PTC-124 and now we are pretty close to real success; As we would define it: making dystrophin and halting or significantly slowing thee disease. We are not giving up on this yet, but my family is disappointed at being so very close and then calling for a STOP; Our son cannot make new "good" muscle, but he has been doing so well on this stuff;
I was on the call and feel very good about PTC. They seemed committed to continue this process. Keep in mind they have a decade's worth of time and resources into this and don't want to see it fail either.
And please don't feel it was for nothing ! (My son has two scars and 18 months of blood draws to show for it !) We have to believe that something can be learned from this. This type of research is so new, that it will take some trial and error!
Try not to be discouraged ! The fight continues !
Tamara Walters, St. Louis
My son would have benefitted from the drug but was too young to take part in the trial. Apparently they have been told in AUstralia to cease the trial immediately as well. My heart is broken. Again. I had a horrible feeling this was going to happen, but kept hoping it was simply me being a pessimist. I have been hoping for this drug ever since I heard about it 3 years ago. Now I am back to no hope as he is almost 6 and realistic treatments are still years away as they have to go through so many years of trials. I am so sad.