AVI Press release - this morning - incredible news for exon 51 skipping!!!

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thanks for the info, great news, cant get here fast enough!
We will have a great 2010...expect the best!!!
I am totally with you. It's really great that the one child did so incredibly well at such a low dose!!!!!!
Have a great holiday and love up those kids of yours. N

jenn said:
thanks for the info, great news, cant get here fast enough!
Without a DOUBT!!!!!!


JUAN PEDRO ARBULU said:
We will have a great 2010...expect the best!!!
This is awesome! My son needs exon 53 skipped, which will hopefully be approved soon after exon 51 is approved.

On a related note - does anyone know if PPMD helps support this project? I don't see it on the website.

Any ideas how we can help push this along? :)
I wish we could get a fire going under, but that red tape just hangs over head. But not for long, I think!!!!!

Keith & Jill VE said:
This is awesome! My son needs exon 53 skipped, which will hopefully be approved soon after exon 51 is approved.

On a related note - does anyone know if PPMD helps support this project? I don't see it on the website.

Any ideas how we can help push this along? :)
Thank you for sharing this information. How exciting! Does anyone have any idea as to how long it takes for the US to get it to people testing? I was aware of the progress being made in the UK and had been tempted to move if need be to get my son the treatment this study seems to be showing such favorable results. I see they are testing in the US via mice. My son has exon 51 skipped. Was diagnosed at 4 is now 6. I pray each day a cure or at least a favorable treatment be found to slow down or best case reverse the progression of DMD. What is the likelihood that the US will be trialing people soon? Curious for those of you who have knowledge in these types of things how this all works in non-scientific terms.
Holding on to hope.
Rhonda
The AVI trial is slated to begin this March - a 12 week study comparing IV delivery to Sub Q. Big Prosensa trial talk of starting this spring as well. Looks to be a much bigger trial with more kids.The UK trials on boys are pretty much complete and have yielded great results with not adverse side effects. Make sure you are registered on all DMD databases as that is where they will call participants from.
I myself thought of going to the UK, but they would not accepts US kids into their trials as they already have their own group of DMD kids. The trials will be here this year - I feel certain.
Hang in there - you are SOOOOOO not alone.
This is going to be a Happy New Year for DMD boys in need to exon 51 skipping.

Rhonda said:
Thank you for sharing this information. How exciting! Does anyone have any idea as to how long it takes for the US to get it to people testing? I was aware of the progress being made in the UK and had been tempted to move if need be to get my son the treatment this study seems to be showing such favorable results. I see they are testing in the US via mice. My son has exon 51 skipped. Was diagnosed at 4 is now 6. I pray each day a cure or at least a favorable treatment be found to slow down or best case reverse the progression of DMD. What is the likelihood that the US will be trialing people soon? Curious for those of you who have knowledge in these types of things how this all works in non-scientific terms.
Holding on to hope.
Rhonda
Curious about registries I may not be aware of. I have Nick signed up on both the DMDRegistry.org via Action Duchenne as well as The United Dystrophinopathy Project from the University of Utah. Are there others out there?

Thanks.
Rhonda


irishgirl said:
The AVI trial is slated to begin this March - a 12 week study comparing IV delivery to Sub Q. Big Prosensa trial talk of starting this spring as well. Looks to be a much bigger trial with more kids.The UK trials on boys are pretty much complete and have yielded great results with not adverse side effects. Make sure you are registered on all DMD databases as that is where they will call participants from.
I myself thought of going to the UK, but they would not accepts US kids into their trials as they already have their own group of DMD kids. The trials will be here this year - I feel certain.
Hang in there - you are SOOOOOO not alone.
This is going to be a Happy New Year for DMD boys in need to exon 51 skipping.

Rhonda said:
Thank you for sharing this information. How exciting! Does anyone have any idea as to how long it takes for the US to get it to people testing? I was aware of the progress being made in the UK and had been tempted to move if need be to get my son the treatment this study seems to be showing such favorable results. I see they are testing in the US via mice. My son has exon 51 skipped. Was diagnosed at 4 is now 6. I pray each day a cure or at least a favorable treatment be found to slow down or best case reverse the progression of DMD. What is the likelihood that the US will be trialing people soon? Curious for those of you who have knowledge in these types of things how this all works in non-scientific terms.
Holding on to hope.
Rhonda
Irishgirl,

I am not really up to date on the research but when you say "great results" what exactly do you mean. From what I have read (and granted that is not a whole lot) they have said that dystrophin has been produced, correct? Have there been any reports regarding improvement in function. I don't know maybe it is just me, or that my son is older and I've been here hearing these "amazing" results for so long with really no functional improvements to the affected individuals. Just wondered if I was missing something.
Those are all good and global! Now, just wait - which is the hardest part of all. My son is missing exons 48, 49 and 50 - so skipping is right up his alley and we closely follow all trial news from Children's here in Washington DC. A big Prosensa trial is scheduled to begin this spring/summer. If you are interested in the AVI trial set to start this March in Ohio, email Ms. Laurence Viollet. SHe is a coordinator of the 12 Ohio trial. Laurence.Viollet@nationwidechildrens.org.
Get on her radar as soon as you can.
Hope this helps. Please let me know if I can help with anything else.
Noreen

Rhonda said:
Curious about registries I may not be aware of. I have Nick signed up on both the DMDRegistry.org via Action Duchenne as well as The United Dystrophinopathy Project from the University of Utah. Are there others out there?

Thanks.
Rhonda


irishgirl said:
The AVI trial is slated to begin this March - a 12 week study comparing IV delivery to Sub Q. Big Prosensa trial talk of starting this spring as well. Looks to be a much bigger trial with more kids.The UK trials on boys are pretty much complete and have yielded great results with not adverse side effects. Make sure you are registered on all DMD databases as that is where they will call participants from.
I myself thought of going to the UK, but they would not accepts US kids into their trials as they already have their own group of DMD kids. The trials will be here this year - I feel certain.
Hang in there - you are SOOOOOO not alone.
This is going to be a Happy New Year for DMD boys in need to exon 51 skipping.

Rhonda said:
Thank you for sharing this information. How exciting! Does anyone have any idea as to how long it takes for the US to get it to people testing? I was aware of the progress being made in the UK and had been tempted to move if need be to get my son the treatment this study seems to be showing such favorable results. I see they are testing in the US via mice. My son has exon 51 skipped. Was diagnosed at 4 is now 6. I pray each day a cure or at least a favorable treatment be found to slow down or best case reverse the progression of DMD. What is the likelihood that the US will be trialing people soon? Curious for those of you who have knowledge in these types of things how this all works in non-scientific terms.
Holding on to hope.
Rhonda
Janine:
You know, they are very hush hush about releasing information. But the one article mentioned a boy who received a very low dose of the drug and produced a high % of dystrophin. In some cases, upwards of 80%. Word is that the drugs work like a charm - even at higher doses. They are also proving to be very safe. They can easily turn a Duchenne into a Becker and BMD folks can live on for quite a while.
How old is your son and how long have you known? I hate being so close to all of this and yet still so far. My son sees Dr. Leshner at Children's here in DC and he is more hopeful than ever before. He has even told us to skip the AVI trial in Ohio in favor of the Prosensa one which will more likely have a test site here in DC. It's a much longer trial and the kids, if the drug works as we all think, will be able to get if afterwards and beyond. My son turned 10 a few months ago and still runs all over the place and is quite strong - so hopefully, we will be making a wise and informed decision to wait.
Janine, I'm scared to death that I will miss something, that is why I post all information I get here. We are together and together we will be stronger and hopefully allow nothing to slip through our fingers. There are some incredibly bright and wonderful people on this site - all in this together. You are in good hands here!!! Noreen

Janine said:
Irishgirl,

I am not really up to date on the research but when you say "great results" what exactly do you mean. From what I have read (and granted that is not a whole lot) they have said that dystrophin has been produced, correct? Have there been any reports regarding improvement in function. I don't know maybe it is just me, or that my son is older and I've been here hearing these "amazing" results for so long with really no functional improvements to the affected individuals. Just wondered if I was missing something.

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