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
Joanna- I'm beyond sad - we were banking on PTC124 for Levi... My hubby was home sick from work today & I simply can't bear to tell him now... I'm so overwhlemed.
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...
To say this news is devastating is an understatement. It seemed to take me back to when we first found out our son had the disorder. Being a scientist myself I know the reality is that there would be more data supporting what they have said already, its just frustrating they havent released it yet for us to see if there is anything that can be pulled from the ashes.