Ace-031 starting the clinical trials for duchenne?

I was just wondering why there are not many response to the ace-031 news. I am personally really excited about this as this is applicable to all and not based on mutation and has the potential to help(buy more time). If they accept US patients, then it is a viable option for folks here in US...

Am I missing something? Is there some news about myostatin inhibition that I am not aware of....just wondering.

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I too am extremely excited about Accelerons ACE-031. I really don't think you are missing anything. My understanding, such as it is, is there was VERY good results in Phase I using healthy post meno women, NO adverse effects & trial results should be coming out soon (May-ish). Keep watching this one. Acceleron just might be able to provide something positive for our guys.

Keeping fingers, toes and eyes crossed while sending daily prayers...must look pretty strange to god :)
I did not get a chance to read it but looks like it has lot of info.
http://www.acceleronpharma.com/content/products/ace-031/faqs.jsp
Thanks amrit and cheryl.
I too am excited about ACE-031 for the fact that it will help all of our boys and SO FAR SO GOOD! Just trying not to get to "trigger happy" after the last few disappointments.
Any body going for the trial?
I sent an email and they are looking into the regulations (if they can accept any US patients)
I agree that this is exciting and would love to hear that another treatment is available.

At the same time, this approach feels a bit like steroids in that it will only give temporary strength. Unless we correct the root cause of our boys' muscle problem, won't the new muscle simply degenerate?

I've tried to read up on this, but haven't found much - so, if I am wrong, please tell me (because I really want to be wrong)!
Yes Steve, there is a lot of discussion about this among scientists and clinicains. The trials will show what happens but that is correct, the new muscles degenerate unless combined with other treatment attempting to "fix/improve" the pathology like gene therapy, exon skipping etc...

Steve Dreher said:
I agree that this is exciting and would love to hear that another treatment is available.

At the same time, this approach feels a bit like steroids in that it will only give temporary strength. Unless we correct the root cause of our boys' muscle problem, won't the new muscle simply degenerate?

I've tried to read up on this, but haven't found much - so, if I am wrong, please tell me (because I really want to be wrong)!
From the Acceleron website..

Treatment with ACE-031 also improves the quality of muscle fibers by reducing fibrosis and decreasing fat infiltration compared to that of untreated animals. ACE-031 also increases expression of utrophin, a protein that is functionally-related to dystrophin and which may compensate for dystrophin deficiency.

I did not know it would upregulate utrophin. This might help the muscles be stronger and not just bigger.

Ofelia Marin said:
Yes Steve, there is a lot of discussion about this among scientists and clinicains. The trials will show what happens but that is correct, the new muscles degenerate unless combined with other treatment attempting to "fix/improve" the pathology like gene therapy, exon skipping etc...

Steve Dreher said:
I agree that this is exciting and would love to hear that another treatment is available.

At the same time, this approach feels a bit like steroids in that it will only give temporary strength. Unless we correct the root cause of our boys' muscle problem, won't the new muscle simply degenerate?

I've tried to read up on this, but haven't found much - so, if I am wrong, please tell me (because I really want to be wrong)!
I agree with the criticism, however since there is so much unknown at the moment, I take anything that can buy us some time. I also found the utrophin up-regulation aspect very interesting.
Have to agree with you Bains. Something, anything, is better than just steriods at toxic levels. I guess the hope for ACE-031 is that even tho our guys won't make dystrophin the ACE-031 will provide an endless (as long as they take it) supply of new muscle fibers. Perhaps then they can walk longer and avoid or delay the other probs that come with being in a chair. Plus, maybe it will replace steriods. That alone would be a tremendous thing. Pretty soon we will all find out more!!
I did not know that. Thanks for posting. If this works as hoped, I expect it to be better than steroids and have less side effects.

Traci Strafuss said:
From the Acceleron website..

Treatment with ACE-031 also improves the quality of muscle fibers by reducing fibrosis and decreasing fat infiltration compared to that of untreated animals. ACE-031 also increases expression of utrophin, a protein that is functionally-related to dystrophin and which may compensate for dystrophin deficiency.

I did not know it would upregulate utrophin. This might help the muscles be stronger and not just bigger.

Ofelia Marin said:
Yes Steve, there is a lot of discussion about this among scientists and clinicains. The trials will show what happens but that is correct, the new muscles degenerate unless combined with other treatment attempting to "fix/improve" the pathology like gene therapy, exon skipping etc...

Steve Dreher said:
I agree that this is exciting and would love to hear that another treatment is available.

At the same time, this approach feels a bit like steroids in that it will only give temporary strength. Unless we correct the root cause of our boys' muscle problem, won't the new muscle simply degenerate?

I've tried to read up on this, but haven't found much - so, if I am wrong, please tell me (because I really want to be wrong)!

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