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Permalink Reply by David on May 21, 2014 at 1:13pm

Yes I'm all too aware that non ambulant subjects will be excluded. It is literally what keeps me up at night.

But 3 months still matters to us...none of this can make it to my son until this phase completes and clock is ticking very loudly

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Permalink Reply by Moein on May 21, 2014 at 1:37pm

I suggest that they include non-ambulant boys and even giving them the priority to keep them move their hands which is the last hope for them.

They can create ameasurement point like grip

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Permalink Reply by David on May 21, 2014 at 2:37pm

Trials at UC Davis in California are establishing these measurements. Don't expect a drug sponsor to take the risk to be among the first to use it tho.

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Permalink Reply by Keith Van Houten on May 21, 2014 at 3:59pm

I don't think the comments about CK drop mean much.  First, they don't say how much it dropped - a lot, a little?  And CK varies quite a bit based on activity.  This is a 12 patient study.  A few of the boys could have had busy days before the baseline measurement and throw the whole thing off.

The positive results here are that the drug shows up in the blood plasma, it's safe enough to enable a phase2 trial, and they've learned that diet has an influence on how the drug is absorbed. 

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Permalink Reply by Michelle Scaglione on May 21, 2014 at 4:28pm

Moein said:

I suggest that they include non-ambulant boys and even giving them the priority to keep them move their hands which is the last hope for them.

They can create ameasurement point like grip

You are so right Moein.  It is their last and only ability towards the end and its heart breaking to see them lose that too.  My son is 21 and is petrified that his hands are going. 

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