In an effort to keep the Duchenne muscular dystrophy community informed, Acceleron and Shire wish to provide an update about the status of the ACE-031 clinical program. During the course of clinical trials in healthy adults and in DMD boys, some participants experienced minor nosebleeds, gum bleeding, and/or small dilated blood vessels within the skin. These events all resolved fully upon discontinuation of treatment. By themselves, the minor bleeding events and dilated blood vessels were not considered to be a serious safety concern for study subjects. However, based on review of these safety data with the FDA and Health Canada, Acceleron has terminated the A031-03 DMD study and has suspended enrollment and dosing in the follow-on extension study A031-06.

Acceleron and Shire remain committed to the global DMD clinical program and the development of ACE-031. To that end, it is our intention to start new studies of ACE-031 in DMD with appropriate safety monitoring following discussions with regulatory agencies. In the coming months, we will provide updates to the DMD community as appropriate.



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so sad and frustrating to see this..

What if all the other trials upcomming this year move with this speed? :(



Ofelia Marin said:

Almost ONE YEAR and nothing from these people. How frustrating is this? Are they still working on this? do they have very clear in mind what 1 year means for our boys?

Ofelia Marin said:

Yes, they are. They look so positive, so frustrating to see that almost a year passed and they did not come out with a solution to minimize the side effects and progress to the next trial. I just cannot believe that our sons need to lose so much time when something with this potential is out there.

David said:

Ofelia - are these the results from the study in Canada that was terminated last year? They seem to be.

If so it makes it all the more baffling that another study has not yet been announced.


The data is nearly identical to what Pat Furlong describes in her blog a few months back, so they've had these results in hand for some time.

I have personally emailed, called and written to both Shire and Acceleron so many times they've stopped responding to me. They will not share any specific information with a parent other than the platitudes that they are "still committed" to this treatment. If PPMD knows anything specific, they can't share it.

I, for one, have stopped contributing $ to any DMD organization until there is news on ACE-031. If they ever do start back up with another trial, that project will get all the $ I've been saving, because these are results like none other I've seen.



 ACE O31 is the most promising treatment at this point and has the potentiality to treat all the boys irrespective of their specific mutation. This is really very frustrating that there is no news to start the trial in coming months.If the side effects are minor then why they are taking such a long time to solve them.

Are there any updates about this drug? It's been 1.5 years since we heard anything. Does Shire still work on this?

Shire came onboard 2 YEARS ago and things just stopped and we did not hear anything from them. Do they still continue development? They even had time to return MDX and produce more data by now if that was FDA required.

I have written many times to someone from Shire, she told me someone would contact me to give me an update but nothing, this was a year ago!

This makes me believe that Shire taking over this drug was not in our son's advantage. So frustrating!


I have written many times to someone from Shire, she told me someone would contact me to give me an update but nothing, this was a year ago!

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