All I have to say about Atalurens update is my son who could of possibly benefited is again left out in the cold.  If and when it ever gets approved it will be too late for my son.

Michelle

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Same here Michelle. I am very sad and upset.

 

I don't understand why all children couldn't participate this time. Its just UNETHICAL. Where are the big organizations in the DMD-field ????? Why can't they prove this to be unethical for all the boys left out in the cold ????? I guess some of the biggest organizations have lawyers in their organization, and I think its about time to use the "compassionate use" rule.  NOBODY seems to bother of all the boys with a nonsense mutation OUTSIDE the trial. I think its a shame and just unethical. 

Well said Berit.  My son missed 2 chances and now is 19 and I can see his decline in the last 3 years that maybe not have been so severe and his and my heart break everyday from duchenne and missed chances.

I agree with you that its time for " the compassionate use". What can we do for this?

 

I would like to hear if Sharon and/or Pat has any comment to this. 

This process is definitely frustrating. I don't see in this article, though, where it says that it will affect the U.S. study in any way...did I miss something somewhere else? It looks like they're just doing what they did in the U.S.-only using boys that had already participated. I realize that some of you are in Europe...and believe me I was pretty upset when they did this here. I have to think it's for a good reason, but I just want my son to have it.

Does anyone at all have any idea what's going on in the U.S. study? Is there any time frame at ALL for when this might be ready for the FDA???

My son is in the trial and we are on a "safety study until approval" is what I have heard. I sometimes wonder if they are just doing this to appease us and they don't think the drug is really working that well. We have been on drug for three years and Zach has declined so much in the past year. He is nine and can hardly walk some days. Don't feel too bad that you did not get on drug, I don't feel it is helping him.

 

However, I have also heard that the drug can be "adjusted" to be more effective, but they have to get the drug approved "as is" before they can look changing it to make it work better. We have been in the trial since the start of phase II and it may still be too late for us anyway.

Tamara

Im sorry to hear that.  Duchenne is slowly killing our sons and we get to sadly and painfully  watch them die.

 

Nonsense friends,

 

I too am waiting for the FDA to approve Ataluren for my son.    I visited PTC in NJ, spoke with Stu the CEO, walked the labs, met the staff and chemists.   Believe me they know the boys are in trouble.  They have pictures of some of the boys in their offices or lab space.   They know time is of the essence.   If they could figure out a way to get it approved tomorrow I am sure they would do it to help save the boys and to also recover some of their huge investments.    Investments both financial and emotional.  They are on our side.   They want what we want.

 

I am sorry some parents think Ataluren may not be working for their son.  Although not a medical pro I studied as much of the data I can get my hands on.   Like any drug their appears to be a range of benefit.   Some appear to have more benefit than others.   We all wish it was a miracle drug - a miracle for all the nonsense boys.   The best I can see from the data is the slowing of progression.  Anecdotally I have heard from reliable sources it appears to almost entirely halt the progression.

 

I don't think PTC doing the 'right' thing by extending the access program to the boys outside the U.S. hurts anyone else's cause.  I suspect it is actually costing PTC money to broaden the access with very little if any benefit as far as supporting the FDA approval process.   In my PTC is being compassionate.

 

I personally think the problem is not PTC.  Rather, I wish Congress would give the FDA or the parents some additional authority to get commercial access to potentially life saving medications under development.   However, from what I understand the FDA has very strict language from Congress.   The process does not seem to be able to handle urgency, rather it seems best at just shuffling along the processess themselves.  While no one wants 'snake oil' I just wish the FDA had some additional latitute to empower the parents and young men.   I think the argument really needs to be made to Congress, not so much the FDA, or least of all PTC.

 

We need to put energy where it will have the most, positive effect.  Demotivation and cynacism travels very quickly.   What are YOU going to do today to fight for your son?  What?   Write it down and then do it.   Think through it.  Call then meet with your Congressman and Senator. Raise money for research - every bit matters.  Encourage other parents, researchers and the doctors.    You're mistaken if you don't think you can make a difference.   Work to save your son and the other boys like him.    Whatever positive work you did, do even more.  Think of all that Pat Furlong has done on your behalf.  Keep working.

 

I am most appreciative of all the work all the parents have put into the fight and I hope I did not offend.

 

Bob

I see your point,Bob, and I am sure PTC is really interested in getting Ataluren approved for all the genetic diseases that might potentially be cured or alleviated by the drug. The problem is that these past two years (2010 and 2011) have been particularly devastating for the DMD community and especially for those of us with sons with nonsense mutations. The way the whole thing about cancelling the trials was handled was terrible, and the absolute lack of common sense that the FDA showed in cancelling the trials just because the primary endpoint "failed" by a margin of les than 1% (30 centimeters out of 30 meters) just blew my mind out and I still can't beleive it, especially considering that almost seven years of prior human trials had shown the drug to be safe. Now they add these additional quite stupid restrictions for the resumed trials and it is impossible not to wonder if the people involved are actually robots.

Aside from that, Do you know, or have they told you at PTC how they're doing with the application for Ataluren in Cystic Fibrosis? I ask this because I have no doubt in my mind that if and when the drug is approved for any disease other than DMD I will do whatever it takes to get it as an off-label drug for my son and give it to him at the low dose that showed to be beneficial.

Of course I respect Tamara's position very much and take her first-hand experience very serioulsy, but in the absence of absolutely anything else, or facing the prospect that the rest of the research avenues might take another decade or so to come to fruition, I just have to cling to this one single, scientifically proven breakthrough that is Ataluren.

Regards

Bernardo 

Sharon, unfortunately we still see no light for non-exonskipping, non-stopcodon boys...something that would benefit all boys (ace31, summit, etc) seem to be going nowhere...

Bob

I believe I have been waiting on Ataluren since 2004.  It has been a long wait with 2 chances of my son to start on the drug and losing the chance due to loss of walking and then it being terminated.  I am not here bashing PTC or anyone else.  I posted a statement and not a question that did not need any response. I certainly do not need you to tell me to fight for my son.  I do that each and everyday and in the end he will die from Duchenne.  My son is 19 not 8.  He is past his chance to live the life he was suppose to have.  You write " I hope I didnt offend anyone"  to write that you knew you would.  I have not lost sight of what Duchenne is doing to my son, maybe you have.  Do not respond to me.  I will not be reading your comment.  I am too busy trying to save my son. 
 
Bob Getler said:

Nonsense friends,

 

I too am waiting for the FDA to approve Ataluren for my son.    I visited PTC in NJ, spoke with Stu the CEO, walked the labs, met the staff and chemists.   Believe me they know the boys are in trouble.  They have pictures of some of the boys in their offices or lab space.   They know time is of the essence.   If they could figure out a way to get it approved tomorrow I am sure they would do it to help save the boys and to also recover some of their huge investments.    Investments both financial and emotional.  They are on our side.   They want what we want.

 

I am sorry some parents think Ataluren may not be working for their son.  Although not a medical pro I studied as much of the data I can get my hands on.   Like any drug their appears to be a range of benefit.   Some appear to have more benefit than others.   We all wish it was a miracle drug - a miracle for all the nonsense boys.   The best I can see from the data is the slowing of progression.  Anecdotally I have heard from reliable sources it appears to almost entirely halt the progression.

 

I don't think PTC doing the 'right' thing by extending the access program to the boys outside the U.S. hurts anyone else's cause.  I suspect it is actually costing PTC money to broaden the access with very little if any benefit as far as supporting the FDA approval process.   In my PTC is being compassionate.

 

I personally think the problem is not PTC.  Rather, I wish Congress would give the FDA or the parents some additional authority to get commercial access to potentially life saving medications under development.   However, from what I understand the FDA has very strict language from Congress.   The process does not seem to be able to handle urgency, rather it seems best at just shuffling along the processess themselves.  While no one wants 'snake oil' I just wish the FDA had some additional latitute to empower the parents and young men.   I think the argument really needs to be made to Congress, not so much the FDA, or least of all PTC.

 

We need to put energy where it will have the most, positive effect.  Demotivation and cynacism travels very quickly.   What are YOU going to do today to fight for your son?  What?   Write it down and then do it.   Think through it.  Call then meet with your Congressman and Senator. Raise money for research - every bit matters.  Encourage other parents, researchers and the doctors.    You're mistaken if you don't think you can make a difference.   Work to save your son and the other boys like him.    Whatever positive work you did, do even more.  Think of all that Pat Furlong has done on your behalf.  Keep working.

 

I am most appreciative of all the work all the parents have put into the fight and I hope I did not offend.

 

Bob

Michelle,

 

I understand you do not want me to respond and you will not read this.   Regardless, I thought I was responding to a discussion group with multiple posts within a discussion thread and addressed my comments broadly (Nonsense Friends), not to an individual or a specific post.  I truly regret any offense.  The last thing I would want to do is create troubles or offense for any other Duchenne parent or boy.

 

I post so seldomly in these boards.  I regret doing such a poor job of posting despite my honest intent of contributing to the discussion and inviting action of the many readers. 

 

I don't know what else to do but apologize once more, learn from the exchange, and apologize once again.

 

Bob

 

 



Michelle Scaglione said:

Bob

I believe I have been waiting on Ataluren since 2004.  It has been a long wait with 2 chances of my son to start on the drug and losing the chance due to loss of walking and then it being terminated.  I am not here bashing PTC or anyone else.  I posted a statement and not a question that did not need any response. I certainly do not need you to tell me to fight for my son.  I do that each and everyday and in the end he will die from Duchenne.  My son is 19 not 8.  He is past his chance to live the life he was suppose to have.  You write " I hope I didnt offend anyone"  to write that you knew you would.  I have not lost sight of what Duchenne is doing to my son, maybe you have.  Do not respond to me.  I will not be reading your comment.  I am too busy trying to save my son. 
 
Bob Getler said:

Nonsense friends,

 

I too am waiting for the FDA to approve Ataluren for my son.    I visited PTC in NJ, spoke with Stu the CEO, walked the labs, met the staff and chemists.   Believe me they know the boys are in trouble.  They have pictures of some of the boys in their offices or lab space.   They know time is of the essence.   If they could figure out a way to get it approved tomorrow I am sure they would do it to help save the boys and to also recover some of their huge investments.    Investments both financial and emotional.  They are on our side.   They want what we want.

 

I am sorry some parents think Ataluren may not be working for their son.  Although not a medical pro I studied as much of the data I can get my hands on.   Like any drug their appears to be a range of benefit.   Some appear to have more benefit than others.   We all wish it was a miracle drug - a miracle for all the nonsense boys.   The best I can see from the data is the slowing of progression.  Anecdotally I have heard from reliable sources it appears to almost entirely halt the progression.

 

I don't think PTC doing the 'right' thing by extending the access program to the boys outside the U.S. hurts anyone else's cause.  I suspect it is actually costing PTC money to broaden the access with very little if any benefit as far as supporting the FDA approval process.   In my PTC is being compassionate.

 

I personally think the problem is not PTC.  Rather, I wish Congress would give the FDA or the parents some additional authority to get commercial access to potentially life saving medications under development.   However, from what I understand the FDA has very strict language from Congress.   The process does not seem to be able to handle urgency, rather it seems best at just shuffling along the processess themselves.  While no one wants 'snake oil' I just wish the FDA had some additional latitute to empower the parents and young men.   I think the argument really needs to be made to Congress, not so much the FDA, or least of all PTC.

 

We need to put energy where it will have the most, positive effect.  Demotivation and cynacism travels very quickly.   What are YOU going to do today to fight for your son?  What?   Write it down and then do it.   Think through it.  Call then meet with your Congressman and Senator. Raise money for research - every bit matters.  Encourage other parents, researchers and the doctors.    You're mistaken if you don't think you can make a difference.   Work to save your son and the other boys like him.    Whatever positive work you did, do even more.  Think of all that Pat Furlong has done on your behalf.  Keep working.

 

I am most appreciative of all the work all the parents have put into the fight and I hope I did not offend.

 

Bob

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