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Seems with these trials it's always two steps forward, one step back. Or sometimes two steps forward, two steps back. Speaking of rumors, I was at an MDA parents meeting last weekend and heard this through the grapevine - now that GSK is taking over the trials they want to eliminate the multi-site approach for the upcoming phase and focus the trial in one location. I was told this location would be in DC. Has anyone else heard this? The person who told me this thought there's a good chance they may back down and return to the multi-site plan, but as of this point doing the trial in DC was now the plan.
Noreen,
I do not think this is true. You can email Dr. Wong directly. I do not believe she could have said that.. it must be a misunderstanding on the part of whomever told you. All mutations "fixable" by skipping 51 should be allowed in the trial.
I'm not that happy about the delay though....
Ofelia
PS There was an exclusion of this type in Jerry Mendell's gene therapy trial, but that is a totally different story...
There are so many rumors flying around. They all need to STOP. I heard nothing like this, in fact I heard quite the opposite from Dr. Eric Hoffman just a few moments ago. The trial for GSK and Prosensa is not moving currently, but is still planned for a bit beyond this spring and will be multi-site. That is the word now from Hoffman, but this is science and science is never perfect and neither are the doctors.
Tapio said:Seems with these trials it's always two steps forward, one step back. Or sometimes two steps forward, two steps back. Speaking of rumors, I was at an MDA parents meeting last weekend and heard this through the grapevine - now that GSK is taking over the trials they want to eliminate the multi-site approach for the upcoming phase and focus the trial in one location. I was told this location would be in DC. Has anyone else heard this? The person who told me this thought there's a good chance they may back down and return to the multi-site plan, but as of this point doing the trial in DC was now the plan.
Ofelia:
Mat just spoke to Eric Hoffman who was completely bewildered upon hearing this insane news. He said there are so many rumors floating around on this. Currently, the trial is not moving because GSK has completely taken over, but it is still set for this year. He could not believe that 48, 49 and 50 would be stripped out. Hoffman said he was going to look deeper into the rumor mill and talk again with Matt this afternoon. So, I will let you know what comes of that conversation.
I hate that a gave into this rumor and I hope that is all it is. But this is not like missing a movie, it's life and death and the person who gave out this information - should be fired as a minimum!!!
Ugh!!!
Talk to you soon,
Noreen
Ofelia Marin said:Noreen,
I do not think this is true. You can email Dr. Wong directly. I do not believe she could have said that.. it must be a misunderstanding on the part of whomever told you. All mutations "fixable" by skipping 51 should be allowed in the trial.
I'm not that happy about the delay though....
Ofelia
PS There was an exclusion of this type in Jerry Mendell's gene therapy trial, but that is a totally different story...
I heard from Dr. Wong's office that they were looking at a late year start on Prosensa, and that beginning hopefully in September. It is terrible hearing one thing and confirming another especially since we're already on pins and needles as it is. I've gotten nothing definitive from Dr. Wong, but because of GSK taking over, that makes sense that her office cannot give specifics because there are none as of yet. I'm just hoping that whatever trial begins, eith AVI or Prosensa, that our boys are accepted and that we can all find a treatment right away. The red tape of the FDA is bad enough as it is.... We've all had to wait on these trials to start, and we've all been holding our breath for something of a miracle, which we've all been researching and could be anywhere from utrophin to stem cells... who knows!! Certainly there has been no true advances that will benefit my son, unless the exon skipping trials go forth as scheduled, and if they can get past the FDA. I'm pretty fed up with the slowness of all of these processes.
Ofelia:
Mat just spoke to Eric Hoffman who was completely bewildered upon hearing this insane news. He said there are so many rumors floating around on this. Currently, the trial is not moving because GSK has completely taken over, but it is still set for this year. He could not believe that 48, 49 and 50 would be stripped out. Hoffman said he was going to look deeper into the rumor mill and talk again with Matt this afternoon. So, I will let you know what comes of that conversation.
I hate that a gave into this rumor and I hope that is all it is. But this is not like missing a movie, it's life and death and the person who gave out this information - should be fired as a minimum!!!
Ugh!!!
Talk to you soon,
Noreen
Ofelia Marin said:Noreen,
I do not think this is true. You can email Dr. Wong directly. I do not believe she could have said that.. it must be a misunderstanding on the part of whomever told you. All mutations "fixable" by skipping 51 should be allowed in the trial.
I'm not that happy about the delay though....
Ofelia
PS There was an exclusion of this type in Jerry Mendell's gene therapy trial, but that is a totally different story...
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