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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'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
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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