This is for the naysayers! Jill you were right! :)
http://ptct.client.shareholder.com/releasedetail.cfm?ReleaseID=518941
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So I guess this is code for "Oops, we make boo-boo, drug good, get going again": Hey we already knew that in 2/2010.
So I guess this is code for "Oops, we make boo-boo, drug good, get going again": Hey we already knew that in 2/2010.
OK... Yes I am extreemly optomistic that Ataluren (aka PTC-124) will be available again for our kids. I know from the trial that our son did very well, though he's no all star, he was in the 2b extension study. So, there is this push for compassionate use via the FDA, and possibility for going to market next year for Cystic Fibroisis where it does work well. My son is 17 now so there is more "need/anger/hope" from me. The 'smart girls and boys' who constructed the trial should have known you don't make muscle, you (that would be impossible) you just slow the degredation. So yeah, really hopeful, angry, about the way things were done, but very, very hopeful.
MichaelMicheal,
Do you have any idea or guess when or if our sons who hwre in the trial will get back on the drug? Weh ave not heard anything from our trial site since the FDA told PTC they needed to do a single tratment IND rather than invidual INDs.
Cheri
Michael Piacentino said:So I guess this is code for "Oops, we make boo-boo, drug good, get going again": Hey we already knew that in 2/2010.
OMG the reply has HTML all over it.OK... Yes I am extreemly optomistic that Ataluren (aka PTC-124) will be available again for our kids. I know from the trial that our son did very well, though he's no all star, he was in the 2b extension study. So, there is this push for compassionate use via the FDA, and possibility for going to market next year for Cystic Fibroisis where it does work well. My son is 17 now so there is more "need/anger/hope" from me. The 'smart girls and boys' who constructed the trial should have known you don't make muscle, you (that would be impossible) you just slow the degredation. So yeah, really hopeful, angry, about the way things were done, but very, very hopeful.
Michael
Cheri Gunvalson said:Micheal,
Do you have any idea or guess when or if our sons who hwre in the trial will get back on the drug? Weh ave not heard anything from our trial site since the FDA told PTC they needed to do a single tratment IND rather than invidual INDs.
Cheri
Michael Piacentino said:So I guess this is code for "Oops, we make boo-boo, drug good, get going again": Hey we already knew that in 2/2010.
Hi Mike,
I am not sure what you mean by HTML but what is your best guess for a time fram as to when our sons will get back on the drug as like you I believe it stabilized Jacob and he is losing ground off the drug.
Thanks, cheri
Michael Piacentino said:OMG the reply has HTML all over it.OK... Yes I am extreemly optomistic that Ataluren (aka PTC-124) will be available again for our kids. I know from the trial that our son did very well, though he's no all star, he was in the 2b extension study. So, there is this push for compassionate use via the FDA, and possibility for going to market next year for Cystic Fibroisis where it does work well. My son is 17 now so there is more "need/anger/hope" from me. The 'smart girls and boys' who constructed the trial should have known you don't make muscle, you (that would be impossible) you just slow the degredation. So yeah, really hopeful, angry, about the way things were done, but very, very hopeful.
Michael
Cheri Gunvalson said:Micheal,
Do you have any idea or guess when or if our sons who hwre in the trial will get back on the drug? Weh ave not heard anything from our trial site since the FDA told PTC they needed to do a single tratment IND rather than invidual INDs.
Cheri
Michael Piacentino said:So I guess this is code for "Oops, we make boo-boo, drug good, get going again": Hey we already knew that in 2/2010.
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