For those who are interested in Gene Therapy please listen to the new podcast release of Dr Kevin Flanigan interviewing Dr Jerry Mendell at

 

http://www.nationwidechildrens.org/muscular-dystrophypodcast

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Quite encouraging. Thanks Cheryl for posting this. Hope FDA gives the green signal quickly.
Is anyone aware of the progress of ACE-031 (of Acceleron)?
You are very welcom & good question Raktim. I've lost track of that approach but it would be nice to find out the status. I'll look when I have more time, next week.

RAKTIM SINGH said:
Quite encouraging. Thanks Cheryl for posting this. Hope FDA gives the green signal quickly.
Is anyone aware of the progress of ACE-031 (of Acceleron)?
ACE-031 is currently testing the compound in healthy folks. LInk below

http://www.clinicaltrials.gov/ct2/show/NCT00952887?term=ace-031&...
Thanks Bains.
Good News,

ACE-031 phase I is finished and results should be posted soon so follow acceleron's website. ACE-031 increased muscles in the post menopausal female healthy volunteers with no adverse side effects. Trials were conducted in Canada. I just returned from a meeting in Newport Beach and the CEO of Acceleron, an exceptionally nice man who expressed his passion to fight DMD, was there. They plan to go forward with DMD clinical testing 2011 for Phase II, hoping for international sites. The sites for phase II have yet to be determined. ACE-031 will not benefit heart or smooth muscles but should increase muscle in all skeletal muscles, which if you think about it really is huge.
Cheryl, we were there as well, I wish I would have had the opportunity to meet you. I had some time with him, and he told Seth and I that a full study of ambulatory boys with DMD will be started being tested within a month in Canada (and yes they already have all the boys for this study)...it is as well so nice to know that Acceleron will not be just looking at the 6MWT rather assessing data via the dexa. He stated that approval in USA has taken some extra time but they just got approval to go through with the study here in the US just last week and they plan on putting the information on the clinicaltrials.gov site within the next month.

This drug sounds so promising. In the meantime has anyone used any of the natural myostatin inhibitors like Celldyne's Folstaxan? It sounds like it is huge in the bodybuilding community and I am curious now after hearing him speak about this.

cheryl cliff said:
Good News,

ACE-031 phase I is finished and results should be posted soon so follow acceleron's website. ACE-031 increased muscles in the post menopausal female healthy volunteers with no adverse side effects. Trials were conducted in Canada. I just returned from a meeting in Newport Beach and the CEO of Acceleron, an exceptionally nice man who expressed his passion to fight DMD, was there. They plan to go forward with DMD clinical testing 2011 for Phase II, hoping for international sites. The sites for phase II have yet to be determined. ACE-031 will not benefit heart or smooth muscles but should increase muscle in all skeletal muscles, which if you think about it really is huge.
Hi Cori,

Haven't heard of the natural myostatin inhibitors. Don't know how or if I should look into them but I suspect with DMD's specialty needs it best to wait for the Acceleron trial to get underway before beginning anything more than steriods (which I HATE). Wish I had a chance to meet you at the meeting...maybe if you live here in Cal we should sked something else?

hugs
cheryl

Cori said:
Cheryl, we were there as well, I wish I would have had the opportunity to meet you. I had some time with him, and he told Seth and I that a full study of ambulatory boys with DMD will be started being tested within a month in Canada (and yes they already have all the boys for this study)...it is as well so nice to know that Acceleron will not be just looking at the 6MWT rather assessing data via the dexa. He stated that approval in USA has taken some extra time but they just got approval to go through with the study here in the US just last week and they plan on putting the information on the clinicaltrials.gov site within the next month.

This drug sounds so promising. In the meantime has anyone used any of the natural myostatin inhibitors like Celldyne's Folstaxan? It sounds like it is huge in the bodybuilding community and I am curious now after hearing him speak about this.

cheryl cliff said:
Good News,

ACE-031 phase I is finished and results should be posted soon so follow acceleron's website. ACE-031 increased muscles in the post menopausal female healthy volunteers with no adverse side effects. Trials were conducted in Canada. I just returned from a meeting in Newport Beach and the CEO of Acceleron, an exceptionally nice man who expressed his passion to fight DMD, was there. They plan to go forward with DMD clinical testing 2011 for Phase II, hoping for international sites. The sites for phase II have yet to be determined. ACE-031 will not benefit heart or smooth muscles but should increase muscle in all skeletal muscles, which if you think about it really is huge.
Great news and thanks for the information Cori and Cheryl.
Trial on DMD kids is going to start within one month (in Canada) which can potentially help all DMD kids!!!!! This is really very good. Pray that it helps ALL.

Are there more news from that newport Beach meeting. Any further info on Molecular Band Aid/Utrophin or exon skipping?
CureDuchenne is planning to post all that was discussed at the meeting and should provide many more details than my sorry brain can produce! You might want to go to their site www.cureduchenne.org in a couple of days Raktim.
best,
cheryl

RAKTIM SINGH said:
Great news and thanks for the information Cori and Cheryl.
Trial on DMD kids is going to start within one month (in Canada) which can potentially help all DMD kids!!!!! This is really very good. Pray that it helps ALL.

Are there more news from that newport Beach meeting. Any further info on Molecular Band Aid/Utrophin or exon skipping?
Raktim, regarding the molecular bandaid, they stated that they thought it sounded pretty promising, however, how well it will work for our boys is questionable. It will bandaid the cardiac muscle, however will not help with the skeletal muscle and the heart has a little bit of skeletal muscle also...It won't help with the lack of dystrophin.

Sounded like with the Exon Skipping that it is looking pretty promising as well. One thing that I got from it was that with alot of the things, like the exon skipping of 51, is that they are hoping that once this gets through clinicals and is shown to work, without any harsh side effects, that they are hoping to get the other exon skipping done WITHOUT all of the clinicals. This is huge! And they believe that is a great possiblity.

It was ALOT of information thrown at us in a short period of time, but I don't remember much indepth talk about the uptrophin other then it is showing positive results and are continuing to work through it as quickly as possible. However to be honest with you, any drug is great, but our son is inline for the 51 exon skip (he has deletion of 52) so I was focused on that...

I hope this helps! Cori

Just as Cheryl stated, CUREDUCHENNE will be posting more info here shortly about the meeting, points made, etc, and a video of the summit as well. Don't know if that will be the beginning of the week or later this week. But keep hoping!

RAKTIM SINGH said:
Great news and thanks for the information Cori and Cheryl.
Trial on DMD kids is going to start within one month (in Canada) which can potentially help all DMD kids!!!!! This is really very good. Pray that it helps ALL.

Are there more news from that newport Beach meeting. Any further info on Molecular Band Aid/Utrophin or exon skipping?
Thanks once again Cheryl and Cori. Loads of promising news. I guess that Newport Beach meeting was really good (in bringing together all DMD experts under roof for discussion for future action plans as well as passing relevant information to DMD families).

Will wait for update on CUREDUCHENNE site.

Raktim

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