Replies to This Discussion

Permalink Reply by Terry Porcaro on June 8, 2010 at 11:08am

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

• ▶ Reply

Permalink Reply by irishgirl on June 8, 2010 at 9:14pm

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

• ▶ Reply

Permalink Reply by Terry Porcaro on June 9, 2010 at 2:11pm

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

• ▶ Reply

Permalink Reply by irishgirl on June 9, 2010 at 2:31pm

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

• ▶ Reply

Permalink Reply by Steve Dreher on June 10, 2010 at 5:29pm

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.

• ▶ Reply

Permalink Reply by Steve Dreher on June 17, 2010 at 11:48am

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.

• ▶ Reply

Permalink Reply by Ofelia Marin on June 17, 2010 at 11:53am

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.

• ▶ Reply

▶ Reply to Discussion

• ‹ Previous
• 1
• 2
• Next ›
• Page