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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We are in STL as well, but have been involved for TWO YEARS. We atill are hopeful that we will learn something from this and continue the fight.

Matt O'Harver said:
We were in the Study at Washington University in MO. And were called this evening and told to quit taking the drug as well. I feel just horrible about what I've put my son through for the past two months and to just be told it's over like that. His biopsy wound still hasn't healed all the way. :(
I agree. Actually for the first test we used a stopwatch with a beep and Zach was walking to the beat of the beep. Then we used on with no beeping ! He always was different depending on how distracted he was !

Matt O'Harver said:
My son is in the non-ambulatory trial, or I guess I should say was. I'm sure it's not a reliable measure of success. But with all the blood work and muscle biopsy's they did, that is where the real results are coming from. I'm extremely heartbroken, my son turned 13 last month and I almost felt like if there was one chance for him, this was it. Now I'm just devastated.

Mindy said:
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...
As we mentioned in yesterday's email, PPMD will be holding an open conference call on Friday, March 5 from 12 - 1pm eastern. This will be an opportunity for you to have your questions regarding the results of the ataluren trial addressed. PPMD President Pat Furlong and Senior Director of Research and Advocacy Sharon Hesterlee will lead the call. We are still awaiting word on whether or not an official from PTC Therapeutics will be able to attend.

For more information on participating in this open conference call, please click here. You will find all of the information you need in order to take part in this important call.
This is exactly how I have been feeling, you hit the nail on the head.

Jonathan said:
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.
With the varibility of the young boys and the 6 min walk it appears even more a reason to continue with the nonambulatory trial and see the endpoints, ie cardiac echo/fx, timed upper body stacking and recall. These older boys do not have the variblity due to attention span ect.

Kim said:
This is exactly how I have been feeling, you hit the nail on the head.

Jonathan said:
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.
In DMD, wouldn't lack of decline be great in itself? Sure improvement is the ultimate goal, but I would take lack of decline as much better than what we have today. I just cannot believe that they can stop this trial before looking at dystrophin expression.


Cheri Gunvalson said:
With the varibility of the young boys and the 6 min walk it appears even more a reason to continue with the nonambulatory trial and see the endpoints, ie cardiac echo/fx, timed upper body stacking and recall. These older boys do not have the variblity due to attention span ect.

Kim said:
This is exactly how I have been feeling, you hit the nail on the head.

Jonathan said:
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.
I agree Ofelia, I heard that the CK levels were dramatically reduced and in turn that would signal a slowing of breakdown of the muscular tissue...although it wouldn't cure the disease, with that one thing alone, I would think that if the CK levels are dramatically reduced, they need to absolutely stay on it....maybe it would give them an extra year or 10 years of walking, which we wouldn't know right from the get-go...but if the CK level are reduced, THE BOYS NEED TO STAY ON IT!!!

My son has a deletion and it wouldn't have worked for him, but I am so so so sad for those who where hopeful regarding this drug, it sounded amazing...keep the faith, I know it is hard right now...it's hard for every single one of us.

Maybe the families that had sons on it need to quit thinking about the drug replacing the dystrophin, because it apparently didn't do what it was intended on doing and fight to see the CK levels, emphasizing that if the CK levels are dramatically reduced, the breakdown is slower...

Stay Hopeful, I know it is hard...Cori

Ofelia Marin said:
In DMD, wouldn't lack of decline be great in itself? Sure improvement is the ultimate goal, but I would take lack of decline as much better than what we have today. I just cannot believe that they can stop this trial before looking at dystrophin expression.


Cheri Gunvalson said:
With the varibility of the young boys and the 6 min walk it appears even more a reason to continue with the nonambulatory trial and see the endpoints, ie cardiac echo/fx, timed upper body stacking and recall. These older boys do not have the variblity due to attention span ect.

Kim said:
This is exactly how I have been feeling, you hit the nail on the head.

Jonathan said:
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.
Please be aware that we will NOT be recording today's open conference call at noon eastern, as previously thought. We have just found out that several officials from PTC will be on today's call and available to answer the questions you have submitted. However, PTC's legal department will not allow the call to be recorded. PPMD will take thorough notes and will distribute these notes within the next few days.
http://www.parentprojectmd.org/site/PageServer?pagename=advancing_r...
Is there anyway to make sure we get those notes forwarded to us. I have meetings all afternoon and cannot attend.

Matt

PPMD said:
Please be aware that we will NOT be recording today's open conference call at noon eastern, as previously thought. We have just found out that several officials from PTC will be on today's call and available to answer the questions you have submitted. However, PTC's legal department will not allow the call to be recorded. PPMD will take thorough notes and will distribute these notes within the next few days.
http://www.parentprojectmd.org/site/PageServer?pagename=advancing_r...
My son was on PTC124 and he is 13 years old and he is already in a wheelchair, so there was no 6 min. walk for him, but
his Phyical Therpist said that he is doing better, the Pulmonary Dr. said that he has stayed the same for over 2 years, he is
stronger then before. He is able to lift his legs, and pick up things off the floor from his wheelchair. I just don't understand and no matter what they tell me this was working. Please dont stop, Please..........
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...
Sharon said the notes would be posted in the next few days.

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