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Ofelia and Christian-
You guys are extremely smart but do you really believe that a treatment, regardless of who own's it, that will snatch a child from certain death would be denied because of inability to pay? Yea, drug companies are in this for profit, but they can't make much if their customers die. I think they know the moral issues at hand. Besides, if they did behave badly I'd like to see them handle a mob of angry parents in a court room...
We've already seen what a judge will do with this issue and PTC124.
cheryl
I dunno about anyone else, but i crack myself up...ugh, too much TV.
"Ofelia and Christian-
You guys are extremely smart"
I dunno about Ofelia, but as for me, I did stay in a Holiday Inn Express last night...
I dunno about anyone else, but i crack myself up...ugh, too much TV.
I am no different than you. I used to ask all the same questions you do. I used to believe 100% in exon skipping.
Where I still believe in it, I believe more in others right now because IF they work, then it would be ready sooner for all DMD patients, not selective percentages. At the rate exon specific treatment is going, it will take what seems like forever to get to all possible skips....and still be leaving 2 behind.
Cheryl,
What I meant is that the cost of the treatment should be a lot LOWER otherwise the pharma companies will not make profits. I am confident that when a treatment becomes reality all the boys will have access to it. Keep in mind that this is a fatal disease (w/o any treatment or cure at this point) so insurance should cover it/part of it.
I also think that they will not be able to approve it only in EU; they need more patients for exon skipping trials, there aren't as many as they need for phase III in EU alone.
Ofelia
cheryl cliff said:Ofelia and Christian-
You guys are extremely smart but do you really believe that a treatment, regardless of who own's it, that will snatch a child from certain death would be denied because of inability to pay? Yea, drug companies are in this for profit, but they can't make much if their customers die. I think they know the moral issues at hand. Besides, if they did behave badly I'd like to see them handle a mob of angry parents in a court room...
We've already seen what a judge will do with this issue and PTC124.
cheryl
"Where does the $250k/treatment number come from."
Paul, I read it as a quote from someone significant to this type of research. I can't recall who, but it was last year when I thought I had what it took to become a researcher (LOL) that I came across it.
It was not just someone on a message board that pulled it out of their woo hoo and threw it up.
I will try to find it again and send you the link....
Hehe. You're too funny Christian! :-)
I agree with Christian. I too have tons of questions and no answers. The sad part is that even the big names in this field, Wilton, Mendell did not have a lot of answers for my questions at this point... I got a "we are on a very steep learning curve right now" from Mendell though. :-)
To be honest, based on what we know at this point, I think that it will take a cocktail (and a good one!) to treat DMD. We should learn more about exon skipping during the next 1-2 years or so.
MicahsDaddy said:"Ofelia and Christian-
You guys are extremely smart"
I dunno about Ofelia, but as for me, I did stay in a Holiday Inn Express last night...
I dunno about anyone else, but i crack myself up...ugh, too much TV.
I am no different than you. I used to ask all the same questions you do. I used to believe 100% in exon skipping.
Where I still believe in it, I believe more in others right now because IF they work, then it would be ready sooner for all DMD patients, not selective percentages. At the rate exon specific treatment is going, it will take what seems like forever to get to all possible skips....and still be leaving 2 behind.
"Ofelia and Christian-
You guys are extremely smart"
I dunno about Ofelia, but as for me, I did stay in a Holiday Inn Express last night...
I dunno about anyone else, but i crack myself up...ugh, too much TV.
I am no different than you. I used to ask all the same questions you do. I used to believe 100% in exon skipping.
Where I still believe in it, I believe more in others right now because IF they work, then it would be ready sooner for all DMD patients, not selective percentages. At the rate exon specific treatment is going, it will take what seems like forever to get to all possible skips....and still be leaving 2 behind.
Hi Ofelia,
I agree with everything you wrote. I wasn't expecting exon skipping to only happen in the UK. If FDA takes another 5-10 years to get "our" version thru I can't wait that long, Alexander can't wait that long. He just turned 10 and by good standards his life is half over. He is not on his death bed right now so if it takes a little more time we might be ok. How many patients do they need for phase III trials - would you happen to know?
cheryl
Ofelia Marin said:Cheryl,
What I meant is that the cost of the treatment should be a lot LOWER otherwise the pharma companies will not make profits. I am confident that when a treatment becomes reality all the boys will have access to it. Keep in mind that this is a fatal disease (w/o any treatment or cure at this point) so insurance should cover it/part of it.
I also think that they will not be able to approve it only in EU; they need more patients for exon skipping trials, there aren't as many as they need for phase III in EU alone.
Ofelia
cheryl cliff said:Ofelia and Christian-
You guys are extremely smart but do you really believe that a treatment, regardless of who own's it, that will snatch a child from certain death would be denied because of inability to pay? Yea, drug companies are in this for profit, but they can't make much if their customers die. I think they know the moral issues at hand. Besides, if they did behave badly I'd like to see them handle a mob of angry parents in a court room...
We've already seen what a judge will do with this issue and PTC124.
cheryl
Hi Cheryl,
I think they need a number in the 100's since DMD is orphan disease...something similar with what they have for PTC124 Phase 2b. Actually I think that they might not even need a Phase 3 if they see significant benefit in boys, Phase 2b would be as far as they need to go in that case.
I too hope they come up with something (anything!) that will buy time for our boys soon. We'll also go to EU or anywhere else if something is approved there faster.
I have some hope in exon skipping since it was applied systemically in dogs and monkeys and they did see improvements and no immune response. I whish they would start this UK trial faster to get some answers.
cheryl cliff said:Hi Ofelia,
I agree with everything you wrote. I wasn't expecting exon skipping to only happen in the UK. If FDA takes another 5-10 years to get "our" version thru I can't wait that long, Alexander can't wait that long. He just turned 10 and by good standards his life is half over. He is not on his death bed right now so if it takes a little more time we might be ok. How many patients do they need for phase III trials - would you happen to know?
cheryl
Ofelia Marin said:Cheryl,
What I meant is that the cost of the treatment should be a lot LOWER otherwise the pharma companies will not make profits. I am confident that when a treatment becomes reality all the boys will have access to it. Keep in mind that this is a fatal disease (w/o any treatment or cure at this point) so insurance should cover it/part of it.
I also think that they will not be able to approve it only in EU; they need more patients for exon skipping trials, there aren't as many as they need for phase III in EU alone.
Ofelia
cheryl cliff said:Ofelia and Christian-
You guys are extremely smart but do you really believe that a treatment, regardless of who own's it, that will snatch a child from certain death would be denied because of inability to pay? Yea, drug companies are in this for profit, but they can't make much if their customers die. I think they know the moral issues at hand. Besides, if they did behave badly I'd like to see them handle a mob of angry parents in a court room...
We've already seen what a judge will do with this issue and PTC124.
cheryl
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