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:
My son was in the study. We were told to discontinue administering the drug. Very sad; heartbroken once again.
Donna, who told you to stop administering the drug? My son is also in the study.
Donna said:My son was in the study. We were told to discontinue administering the drug. Very sad; heartbroken once again.
Our study site called me today.
Joanna Johnson said:Donna, who told you to stop administering the drug? My son is also in the study.
Donna said:My son was in the study. We were told to discontinue administering the drug. Very sad; heartbroken once again.
Joanna I feel the exact same way. We were at CHOP for Phase 2b and in Boston since July for the extension. I got a voicemail message from Boston but haven't heard back yet.
He was doing so very well. He is almost 11. I just don't understand.
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