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Exon 51 skip (GSK2402968) in ambulant boys Canada

Phase III GSK exon 51 skip ambulatory boys, recruiting.
48 week trial, Montreal, we are starting soon.

weekly injection (subcutaneous), 2 muscle biopsies, etc,etc.

Here we go!

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Permalink Reply by Ofelia Marin on June 8, 2011 at 4:12pm

Good luck!

Permalink Reply by Andrew Kerr on June 8, 2011 at 6:24pm

Best of luck to you. Its nice to see exon skipping in Phase III trials.

Is this a blind trial, or do you know that they'll be getting the drug? Love to hear how it goes, please keep us informed.

Permalink Reply by Angela Bourgeois on June 8, 2011 at 6:34pm

Andrea,

Best of luck! Our son had the double biopsy, blood draws (no injections, though), diaries and the six minute walk test for the phase 2b PTC 124 trial. Your son will definitely get tired of watching that 6 minute walk video (lol). It was worth it all though. Ever though Jacob was on the placebo during the double blind trial he is now getting the drug through the open label safety trial. Hoping they all work and some day we will have a choice of treatments!! Wouldn't that be amazing! We are so close!

Angela

Permalink Reply by Andrea Cleary on June 8, 2011 at 8:36pm

@ Andrew Double blind study, but would get the drug in extension study after 48 weeks.

Permalink Reply by Clare Harper on June 9, 2011 at 8:17am

best of luck !!!! Keep us all posted!!

Permalink Reply by Liisa Underwood on June 9, 2011 at 11:53am

Rock on Andrea!!!! I'm praying for this to work for our boys!

Permalink Reply by Lindy Wilhelm on June 9, 2011 at 9:25pm

Praying our day has come!

Permalink Reply by Jennifer on July 8, 2011 at 3:15pm

Does anyone have any contact info for the trial site in Montreal? I have a number (514-345-4931 ext 6729), but I can't seem to connect with anyone there who speaks English well enough, so I get transferred to someone who knows nothing about the trial.

Thanks,

My email address is metcalfefamily@hotmail.com

Jennifer

Permalink Reply by Andrea Cleary on July 8, 2011 at 5:53pm

Jennifer,

I just e-mailed your above address with some info.

Andrea

Permalink Reply by Bryan & Cari Sweet on July 8, 2011 at 9:33pm

Hi Andrea, you wouldn't mind sending me that info too?

Thanks

Bryan

Permalink Reply by Andrea Cleary on July 8, 2011 at 11:09pm

When I said "here in Montreal", I meant as a trial site for the Eastern part of Canada.

Andrea Cleary said:

@ Angela Yes, so close that I can taste it, but still worried it's too late for him, ya know? I wish he was younger (11 in July)

They want to get 6 boys here in Montreal, so 4 would get the drug, and 2 the placebo.

Permalink Reply by Paul Cliff on July 22, 2011 at 4:13pm

Has anyone seen the full text of this article?

http://www.nejm.org/doi/full/10.1056/NEJMoa1011367

If the data is as good as the abstract, where it claims a mean increase of 10% in the six minute walk test after the 12 week extension, then this should be some good stuff. I'd be curious as to what the mean change in a six minute walk test is after 12 weeks for the general DMD population in any event.