Dr. Diana Escolar is back and her trial ACE-031 will be underway soon - and there will be five sites in the US for the next trial.  FDA loves the trial.  Cannot wait for it.  It may beat Prosensa (GSK) trial here.  

Yesterday, she began seeing patients again, only four per month so she can devote the rest of her time to research.  SHe is completely excited about this trial.

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I'm a little confused and having a hard time folowing this thread. ACE-031 is a myostatin inhibitor, so the muscle will be allowed to grow. How is that connected with completing the code for those who need exon 51 to be skipped?

irishgirl said:
This particular trial of ACE-031 is for those 4 and older with confirmed DMD diagnosis. While it does all you say, it completes the code for those need exon 51 to be skipped. There is also a trial for it with premenopausal women. Just nice to have three treatments to look forward to - instead of nothing - the case for so many years. I feel really good about all of this. None of these are cures and they were never professed to be, but a plausible treatment to get our kids to the next step.

Michelle said:
http://www.acceleronpharma.com/content/products/ace-031.jsp

ACE-031 isn't just for DMD. It's a muscle building compound for muscle wasting diseases in general. It doesn't cure DMD or assume to. It just builds muscle. In our case, it's more of a race against time. Build muscle in the hopes of delaying the tissue degeneration.

Karen flor said:
Hi! I have read many things about both the Prosena {exon skipping 51} and ACE-031 but need help!! Comparing the two, what are the advantages of one over the other or are there???? From what I understand ACE-031 can be used for a wider scope of boys than exon skipping 51. However, my son fits into the exon skipping 51 group and I'm hoping to get him into a trial, but would like to know if I'm barking up the wrong tree and the ACE-031 might be a better treatment. Any thoughts???
Karen
I apologize if I caused any confusion - I meant that ACE-031 builds muscle the way exon skipping completes the DNA strand code. While they are different drugs, they can both help our boys. I was a bit overzealous in my words and was in a hurry as I wrote.


Terry Porcaro said:
I'm a little confused and having a hard time folowing this thread. ACE-031 is a myostatin inhibitor, so the muscle will be allowed to grow. How is that connected with completing the code for those who need exon 51 to be skipped?

irishgirl said:
This particular trial of ACE-031 is for those 4 and older with confirmed DMD diagnosis. While it does all you say, it completes the code for those need exon 51 to be skipped. There is also a trial for it with premenopausal women. Just nice to have three treatments to look forward to - instead of nothing - the case for so many years. I feel really good about all of this. None of these are cures and they were never professed to be, but a plausible treatment to get our kids to the next step.

Michelle said:
http://www.acceleronpharma.com/content/products/ace-031.jsp

ACE-031 isn't just for DMD. It's a muscle building compound for muscle wasting diseases in general. It doesn't cure DMD or assume to. It just builds muscle. In our case, it's more of a race against time. Build muscle in the hopes of delaying the tissue degeneration.

Karen flor said:
Hi! I have read many things about both the Prosena {exon skipping 51} and ACE-031 but need help!! Comparing the two, what are the advantages of one over the other or are there???? From what I understand ACE-031 can be used for a wider scope of boys than exon skipping 51. However, my son fits into the exon skipping 51 group and I'm hoping to get him into a trial, but would like to know if I'm barking up the wrong tree and the ACE-031 might be a better treatment. Any thoughts???
Karen
Thanks for the clarification. I admit, I was getting a little confused there for awhile.

irishgirl said:
I apologize if I caused any confusion - I meant that ACE-031 builds muscle the way exon skipping completes the DNA strand code. While they are different drugs, they can both help our boys. I was a bit overzealous in my words and was in a hurry as I wrote.


Terry Porcaro said:
I'm a little confused and having a hard time folowing this thread. ACE-031 is a myostatin inhibitor, so the muscle will be allowed to grow. How is that connected with completing the code for those who need exon 51 to be skipped?

irishgirl said:
This particular trial of ACE-031 is for those 4 and older with confirmed DMD diagnosis. While it does all you say, it completes the code for those need exon 51 to be skipped. There is also a trial for it with premenopausal women. Just nice to have three treatments to look forward to - instead of nothing - the case for so many years. I feel really good about all of this. None of these are cures and they were never professed to be, but a plausible treatment to get our kids to the next step.

Michelle said:
http://www.acceleronpharma.com/content/products/ace-031.jsp

ACE-031 isn't just for DMD. It's a muscle building compound for muscle wasting diseases in general. It doesn't cure DMD or assume to. It just builds muscle. In our case, it's more of a race against time. Build muscle in the hopes of delaying the tissue degeneration.

Karen flor said:
Hi! I have read many things about both the Prosena {exon skipping 51} and ACE-031 but need help!! Comparing the two, what are the advantages of one over the other or are there???? From what I understand ACE-031 can be used for a wider scope of boys than exon skipping 51. However, my son fits into the exon skipping 51 group and I'm hoping to get him into a trial, but would like to know if I'm barking up the wrong tree and the ACE-031 might be a better treatment. Any thoughts???
Karen
When I hear about new things that can be positive treatments - I get very excited and tend to write too quickly.
I am just thrilled by there being three very promising treatments for my son and many others.


Terry Porcaro said:
Thanks for the clarification. I admit, I was getting a little confused there for awhile.

irishgirl said:
I apologize if I caused any confusion - I meant that ACE-031 builds muscle the way exon skipping completes the DNA strand code. While they are different drugs, they can both help our boys. I was a bit overzealous in my words and was in a hurry as I wrote.


Terry Porcaro said:
I'm a little confused and having a hard time folowing this thread. ACE-031 is a myostatin inhibitor, so the muscle will be allowed to grow. How is that connected with completing the code for those who need exon 51 to be skipped?

irishgirl said:
This particular trial of ACE-031 is for those 4 and older with confirmed DMD diagnosis. While it does all you say, it completes the code for those need exon 51 to be skipped. There is also a trial for it with premenopausal women. Just nice to have three treatments to look forward to - instead of nothing - the case for so many years. I feel really good about all of this. None of these are cures and they were never professed to be, but a plausible treatment to get our kids to the next step.

Michelle said:
http://www.acceleronpharma.com/content/products/ace-031.jsp

ACE-031 isn't just for DMD. It's a muscle building compound for muscle wasting diseases in general. It doesn't cure DMD or assume to. It just builds muscle. In our case, it's more of a race against time. Build muscle in the hopes of delaying the tissue degeneration.

Karen flor said:
Hi! I have read many things about both the Prosena {exon skipping 51} and ACE-031 but need help!! Comparing the two, what are the advantages of one over the other or are there???? From what I understand ACE-031 can be used for a wider scope of boys than exon skipping 51. However, my son fits into the exon skipping 51 group and I'm hoping to get him into a trial, but would like to know if I'm barking up the wrong tree and the ACE-031 might be a better treatment. Any thoughts???
Karen
Hello All,

There was an earlier discussion of ACE-031 here: http://duchennecommunity.ning.com/forum/topics/ace031-starting-the-... -

One thing to keep in mind - according to the company's literature, in addition to inhibiting myostatin, ACE-031 may upregulate utrophin - sounds pretty amazing if it lives up to the claims. It certainly would change many of our sons' treatment regimens.
Hello -

Can anyone put me in touch with Dr. Escolar, or tell me where I might read specifically about her research? I am interested in donating to support her research, but not sure how to do that. Thanks.
She is on Facebook.

Steve Dreher said:
Hello -

Can anyone put me in touch with Dr. Escolar, or tell me where I might read specifically about her research? I am interested in donating to support her research, but not sure how to do that. Thanks.

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