My sons is 4 yrs 9 months. His deletion is big (3 to 43).  Before knowing the deletion I was encouraged about exon skipping.  I asked Dr Wilton from Australia if his deletion could be helped, he said probably not.  It seems like so much research is going in this direction.  Do we have any reason to help in other areas.  We are losing hope and faith.  Please respond.

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My son had a clinic visit few days ago, where our doc described some very positive messages coming out of Acceleron regarding ACE-031 trial in Canada. This study is very small, and only for ambulant boys. Normally his doc is very, very cautious about making such statements, and he was quick to clarify the study won't complete until Feb 2012 ... but his body positive language and excitement were impossible to miss.

 

This treatment would apply to all boys, irrespective of deletion. Here's to hope.

one question

1) phase II wont complete until Feb2012, or whole clinical trial process?

if i am correct this trial is fast tracked, i am just trying to understand when you to expect the drug as therapy?

thanks.

David said:

My son had a clinic visit few days ago, where our doc described some very positive messages coming out of Acceleron regarding ACE-031 trial in Canada. This study is very small, and only for ambulant boys. Normally his doc is very, very cautious about making such statements, and he was quick to clarify the study won't complete until Feb 2012 ... but his body positive language and excitement were impossible to miss.

 

This treatment would apply to all boys, irrespective of deletion. Here's to hope.

So this small dosing trial started in 2010 and will end in 2012? Wow, I did not expect this. They are moving incredibly slow. In general 2 year duration happens in a large pivotal trial. Very dissapointing. Positive results or not, last year they said that will expand in US this year and it's already April and we did not hear anything about start of US trial.

 

Amrit, this is only a dosing trial. It needs to be followed a large efficacy multi-center trial with a duration of 1 year, hence

~2 years including enrollment etc.

David said:

My son had a clinic visit few days ago, where our doc described some very positive messages coming out of Acceleron regarding ACE-031 trial in Canada. This study is very small, and only for ambulant boys. Normally his doc is very, very cautious about making such statements, and he was quick to clarify the study won't complete until Feb 2012 ... but his body positive language and excitement were impossible to miss.

 

This treatment would apply to all boys, irrespective of deletion. Here's to hope.

Thanks Ofelia for making it clear for me. it is moving slow. Not sure what fast track mean? :(
I have heard that the American trial will begin by year end, but will be for steroid naive boys.  They are doing this to see what dosing is best...with or w/o steroids.  I don't like it, but they didn't ask me! UGH
That would make in a very slow enrollment...

Lori Ware said:
I have heard that the American trial will begin by year end, but will be for steroid naive boys.  They are doing this to see what dosing is best...with or w/o steroids.  I don't like it, but they didn't ask me! UGH

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