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Permalink Reply by Ofelia Marin on December 29, 2009 at 8:25pm

Janine,

It is too early to know the extent of functional improvement. Next year will be very important for exon skipping. This is the only place where I read about "early signs of functional improvement":http://community.parentprojectmd.org/profiles/blogs/notes-from-trea...

Pat's summary for Prosensa's drug clinical benefit says:

"Clinical effects: after 5 weeks. (patient has not reached steady state) trends in functional improvement and drop in CK. In the highest dose groups, there is a trend toward decreased CK levels and early signs of functional improvement as determined by 6 minute walk test and functional testing.

There is some confidence that with longer studies, they will be able to demonstrate safety and efficacy."

I hope this will be proven next year. Very important to know if there will be functional improvements in ALL boys in the trial or only some. It looks promising so far.





Janine said:

Irishgirl,

I am not really up to date on the research but when you say "great results" what exactly do you mean. From what I have read (and granted that is not a whole lot) they have said that dystrophin has been produced, correct? Have there been any reports regarding improvement in function. I don't know maybe it is just me, or that my son is older and I've been here hearing these "amazing" results for so long with really no functional improvements to the affected individuals. Just wondered if I was missing something.

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Permalink Reply by Colleen on December 29, 2009 at 8:39pm

That is very exciting! Can't wait to hear more!

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