Deletion of exon 51

For the parents of boys with a deletion of exon 51.

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Latest Activity: Jan 24, 2016


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Comment by christine good on August 12, 2009 at 9:19pm
I don't think this applies to us, Julie. With a deletion of 51, you need 50 skipped.
Comment by Julie Hathaway on August 12, 2009 at 9:06pm
Where did you sign up for Dr. Mendel's trial? I didn't know you could do that just yet. Tell me more about the trial in the Netherlands. My email is Thanks so much.
Comment by Michelle Gonzales on August 12, 2009 at 1:33pm
My Nicolas has deletions of 45-50 and he sees Dr. Wong. A month or 2 ago, I signed up for Dr. Mendel's trial for the clinical trial for AVI. I spoke to Dr. Wong about it and she said that she will be doing the Netherlands study, which is Prosena (not sure if spelling it correctly), and that they have seen better results with Prosena. She wasn't putting AVI down at all, just basically said that it was in the beginning stages and Prosena has been tested further.

Not sure when these trials will begin here. Nicolas is already 7 years old and am afraid with the way the very slow process is with any and all trials, this will not benefit him at all. Hopefully we hear something VERY SOON!
Comment by Julie Hathaway on June 7, 2009 at 9:42pm
Tell me more, Christine. Will you be at the conference in June?
Comment by christine good on June 7, 2009 at 2:18pm
Hi all,
This one is for us. God Bless.

AVI BioPharma Signs an Additional Drug Development Contract with Charley’s Fund Inc. for Duchenne Muscular Dystrophy
Comment by jenn on April 10, 2009 at 7:10am
thanks, i went to the avi web site that someone posted, it does look promising though, with exon 50 being next... i also went to the trial recruiting site, and read the requirements for participation in trials. the only part that scares me is that boys must be able to walk to be in the next trial, they better hurry for austin! he's 10 and can walk some, but not for long i suspect
Comment by christine good on April 10, 2009 at 12:30am
I went through this a few months ago. It took awhile to get through my thick skull, but this trial does not include us at this time. Its oxymoronic, but they need 50 to be skipped. We are next on the totem pole though.
Comment by jenn on April 9, 2009 at 8:58pm
so, the newest quest magazine came out today, with a glowing exon skipping article ( i know we have all heard about the trial, it was just the first time i saw it in print) i have to say i let myself get a little excited, for the first time in a long while. my question is, does anyone understand if skipping exon 51 treats kids with an exon 51 deletion, i have heard some of you say no, why not? and also any idea how we get more info on who is doing the next step, and how do we get involved? i have not been to the conference since 2007 and it looks like despite our best attempts we may not have the available cash or childcare to go this summer ( still hopefull though!) any insight would be great, thanks.
Comment by Karen Barnett on February 22, 2009 at 4:32pm
Exon 51 trials did already start in England (Dr. Muntoni) and the Netherlands (Leiden) last year. This will help the largest majority of boys with DMD who have (I believe) the deletions 45-48 Charley's Fund donated 2.5 million to AVI in 2007 to get research going and hopefully a trial in 2010 for skipping exon 50.
Comment by christine good on February 22, 2009 at 4:17pm
I check these guys pretty regularly, plus there is a thread on PPMD main page. It already started in England.

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