I heard the Dr. Wong has said that the upcoming trial of Prosensa's exon 51 skipping will be EXCLUDING all children with deletions of exons 48, 49 and 50. I cannot believe this - and I don't know how it could be possible.

Does anyone have any news on this??? S.O.S.

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I just spoke to Steven with Treat-NMD is New Castle, UK. He said that since the Prosensa merge with GSK, GSK will be taking over the trial. Currently, the trial is not moving forward. It is still planned for the Spring of this year, but will be pushed back a little, but is still looking at a 2010 start. Steven also said that those with deletions of exons 48, 49 and 50 WILL BE AMENABLE to the PRO051. The current trial plan will be to include those missing 48, 49 and 50. It's what they have worked for.
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...
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.
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 agree - relying on all the whispers and rumors floating around gets very frustrating. Especially when you hear things that directly contradict one another. Is there anywhere an official statement on the current state of planning for the trials? I understand the hesitancy to say anything definitive while plans are being hammered out, but I think any kind of official update would be appreciated by all of us and hopefully lay to rest unwarranted rumors. It seems right now all our information is coming from this person who spoke with that person, etc.


irishgirl said:
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.
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.
Right...ridiculous rumors...Don't worry about them. They do not make any sense.

It makes complete sense to me that there might be a little delay if GSK is planning Phase III, hopefully not too long.

irishgirl said:
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...
This is one other thing. I think we need to stop listening to each individual doctor who tells his/her opinion. Unless they are directly involved in the planning of the trial, they do not know the exact timing.

GSK will make the official announcement when the time comes. No patient will be enrolled before GSK makes the announcement. If Dr. Wong is not 100% sure that Cincinnati is one of the trial sites, then there is now way she knows exactly when the trial starts. We just need to be patient...easy to say, hard to do!





Michelle Gonzales said:
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.
Noreen, you are not kidding. There are real boys lives on the line here. My heart almost stopped when I read the first post on the discussion, thank goodness the doctors in charge and giving us the right information. I try to listen to what I hear directly from the doctors. It's so hard, I just want to wake up one day and someone say, "Hey give Wyatt this treatment and his worries are over!" We just as a :PPMD community need to stay strong and talk to each other. We will win this fight, we just need patience and that's pretty impossible for me. I want to fix it now.

irishgirl said:
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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