Ataluren Results Announced

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

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Very sad news :(
I appreciate you bringing this to our attention Pat. I was hoping for a different outcome even tho Ataluren wouldn't work for my son anyway. Sure wanted somebody's son to escape this mess. :( :( :(
My son was in the study. We were told to discontinue administering the drug. Very sad; heartbroken once again.
It wouldnt have helped Justin, but I had such hopes for the boys it would help. My heart is broken.
My son would not benefit either from this but am saddened as well!
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.
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.
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.
My sons would not benefit either from this but am saddened as well!
We're supposed to have a conference call with our dr. tomorrow. We are at CHOP in Phila. Both of my sons have Duchenne, my only 2 children. I feel like I am back to the day we got their diagnosis. I am sick to my stomach and helpless once again. Afraid to hope, scared to breath. Worried what will come next...

Donna said:
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.
We felt that Elliott was doing so well too. I don't know what to think. My mind is racing in 1000 crazy directions.

Angela Bourgeois said:
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.
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.

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