Dear Friends,
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.
Sincerely,
Pat Furlong
Founding President and CEO
Tags:
I hope that the call will be summarized by someone on the boards afterwards for those of us who can't get in because the call is full...
I'll email you.
Mindy said:I hope that the call will be summarized by someone on the boards afterwards for those of us who can't get in because the call is full...
Do the rest of you all feel like the walking test is a reliable measure of success? With my son, I could see the results of the walk test varying significantly by the day just based on his mood alone...
Nick Catlin from Action Duchenne in the UK has put somes notes of his own from the meeting on the Action Duchenne website:
http://www.actionduchenne.org/viewarticle?news=53
best wishes
Mark
After reading this, I'm a bit upset at PTC. I feel they have an obligation to the DMD community and to those families that have helped them during this trial. The question of continuing the trial would be hard because they would have to ask regulators for permission? Wow thats very arrogant when you are dealing with our children's lives. If they have any sort of proof that a number or even 1 boy has been helped in any way they need to fight to continue the trial. If CPK levels have dropped, dystrophin made, or even just slowed down the progression it's more then we have now. My son was only allowed on the medicine for 4 weeks, he now has a big scar on his leg and has been through numerous blood draws, and essentially for nothing because the pulled the plug on all of this. I want some real answers and some real data put out on the table for our sons. They owe that to the DMD community.
Marks33 said:Nick Catlin from Action Duchenne in the UK has put somes notes of his own from the meeting on the Action Duchenne website:
http://www.actionduchenne.org/viewarticle?news=53
best wishes
Mark
Notes from the call will go up on Monday. NO ONE (not this community and not PTC) is giving up on Ataluren. The sub analysis is underway. We are learning and will learn a great deal more in the upcoming weeks and months. This is a roadblock and we have to figure out how to get around it and we will. We want the best way forward, the 'way' that has the greatest chance of success for every boy/young man who has the potential to benefit from this approach.
Notes from the call will go up on Monday. NO ONE (not this community and not PTC) is giving up on Ataluren. The sub analysis is underway. We are learning and will learn a great deal more in the upcoming weeks and months. This is a roadblock and we have to figure out how to get around it and we will. We want the best way forward, the 'way' that has the greatest chance of success for every boy/young man who has the potential to benefit from this approach.
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