Are their any open clinical trials for deletion 22-41?

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Yes, thanks Jason. But since this trail is i Canada, how is FDA involved? Sorry if I am asking a silly question. I have been hoping for US trails to begin.

yes it is a Canadian trial but from what I'm being told it is being monitored by the FDA for future approval consideration.   So if Accererlon wants the FDA to accept the results they need to allow their input.  

At least that's my understanding of what I was told  :)

And this being a dosing trial, I'm assuming all the data obtained in the Canadian trial is part of the package sent to the FDA for approval of start of US trials.

 

There was a lot of noise about this trial last year before and after Shire came on-board... we did not hear anything for a while now.

Hi,

Suppose that ACE 031 is effective in muscles but causes nose bleeds,so in that case will this drug be approved based on a fact that Steroids cause many serious side effcts but they are approved in DMD?!

Can anybody elaborate on this?

Hearing about the nose bleed i am disappointement and losing hope. I hope i am wrong and they can figure something out please please....

You know,I am full of hope,and I feel that this disease will be under control soon.

I heard that nose bleeds is just in those who receive high dose

Thanks Moein, your comment really brigthen me. My son is getting older and really want something soon.
The only effect we saw, positive or negative, from ACE31 was nosebleeds.   I'd like to think it has the potential to help but so far we've seen nothing.

As for the nosebleeds... they were in the second cohort. 

The dosings were 0.5, 1.0, ....and 2.0 which was to be lowered to 1.5 and hasn't started due to the hold.

 

2.0 was what the healthy adults were given. 

Do you mean that boys who are enrolled in ACE 031 WERE GETTING NO BENEFITS?
They did not get to the high dose group. They only completed the 2nd cohort from what I recall. Jason, have they completed the 3rd as well at 1.5 instead of 2?

Moein said:

You know,I am full of hope,and I feel that this disease will be under control soon.

I heard that nose bleeds is just in those who receive high dose

We are not privy to any of the results except our own, so I have no idea how other boys responded.   I just know that we didn't really see any functional improvement.   His strength and function testing seemed to remain consistent. 

He was only on it for the initial three months so who knows if we will better results longer term. 

 

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