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Permalink Reply by christine good on April 25, 2009 at 7:41pm

I emailed Lindsay and she replied immediately. They are gathering named for the database and she says that we definately qualify (del 51). She says that it will start sooner, rather than later.

Christine

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Permalink Reply by Karen Barnett on April 25, 2009 at 9:20pm

Well, that's a surprise seeing as we need exon 50 skipped for our boys with a deletion of 51.

christine good said:

I emailed Lindsay and she replied immediately. They are gathering named for the database and she says that we definately qualify (del 51). She says that it will start sooner, rather than later.

Christine

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Permalink Reply by jenn on April 25, 2009 at 9:40pm

according to the website my boys also qualify, but it does say 51 does not... lindsay wrote us back too, and said that we will be on the recruiting list, im not sure what if anything that means, now im confused

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Permalink Reply by Darcy Tumminello on April 25, 2009 at 9:42pm

I'm a little confused. My son has deletion of 51-55. Shoul dI be emailing to get on the list? Sounds like it?

Darcy

Karen Barnett said:

Well, that's a surprise seeing as we need exon 50 skipped for our boys with a deletion of 51.

christine good said:

I emailed Lindsay and she replied immediately. They are gathering named for the database and she says that we definately qualify (del 51). She says that it will start sooner, rather than later.

Christine

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Permalink Reply by christine good on April 26, 2009 at 12:02am

Karen,
They are starting preclinicals for 50 as well as many other deletions. I checked the AVI website.

Christine

http://www.avibio.com/duchenne_muscular_dystrophy.php

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Permalink Reply by Karen Barnett on April 26, 2009 at 12:11am

Are they recruiting (or taking names) for 50 right now with Dr. Mendell as well? If so, I'll be on the phone to them as well first thing on Monday.

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Permalink Reply by christine good on April 26, 2009 at 2:01am

The person that wrote back siad this:

Mrs. Good,

Thank you for contacting me. It does appear the Kian's deletion would qualify him to be on the recruitment list. If you could send me his name, date of birth, address and a phone to reach you, I will get him in the database today. Right now we are in the recruitment phase. We are optimistic that this trial will begin sooner, rather than later. I will be in touch with all the families as the process progresses, with more details.

All the best,
Lindsay Arnott

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Permalink Reply by irishgirl on April 27, 2009 at 10:49am

Hi:
This new trial will be injection only - delivered to a particular muscle. They want to up the dose. We already know the injections work, but one cannot inject the entire body - it has to be done systematically through IV delivery to the entire body - especially the heart. You take part in this trial and will potentially see great results - but only in that particular muscle. And when the trial is over, so are the potential results. These are just studies - not an actual treatment that will occur in the next few months after the trial. If you look at all the inclusion/exclusion criteria - one stipulation says that your may not have participated in a similar trial during the past year. Keeping in mind that each trial will have their own set of inclusion/exclusion rules, but generally, if you are on one trial, you will not just blend into the next where the dose will be higher and yield even greater results. Get all the facts and have all your ducks in a row before jumping into a trial - especially if a similar trial has been done before. Again, it is a trial and not a treatment. But I am convinced that the IV delivery - yielding tremendous results - is right around the corner and will come to fruition in time to help young boys helped by skipping exon 51.

Michelle Gonzales said:

Thank you Mindy!! My son qualifies for exon skipping and we see Dr. Wong. She had our hopes up last year when she asked us if we wanted to be part of systemic trials this summer. Earlier this year, I emailed her for update and she stated that trials are only in Europe. I deflated at that time because I was so full of hope that my son could be part of trials now, and hopefully for all our boys help curb this disease - or, in the very least, help the boys of the future.

After reading your discussion.... I'm floating in the air again, just with the thought that this could really help my son as well as the many others out there!!

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Permalink Reply by Mindy on April 27, 2009 at 12:28pm

Hello all,
I think we all need to be careful about a couple of things:
1. These trials are very preliminary right now. The clinic has not met with the FDA to establish protocol, final qualifications, etc. I don't think we should speculate on any of that information until it is finalized so that we don't spread rumors. Lindsay has said that she'll be in touch with the families personally with any developments, so if you hear anything from anyone other than her, don't take it as the final word.
2. We need to be respectful of the people planning these trials, including being careful about posting emails from them, etc. without their permission. Trials like these are very fragile in the planning stages, and agitating in any way doesn't help the process.

IHere are the facts of the trial from Lindsay:
it is systemic, not intramuscular
we don't know dosage
there are risks involved in participating and no guarantees

'm not trying to be the cop here, but I don't want Mendell's office bombarded with controversy and conflict when they're just trying to assess interest in participation.

Thanks,
Mindy

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Permalink Reply by Kelly Provenzano on April 27, 2009 at 2:33pm

Thanks Mindy:
I agree and lets face it - we as parents are going to get the proper information before we enroll our children in a trial. We know that there has to be more information and that this will come in time from the proper people - but maybe we can take a break from the desparation we feel and enjoy the hope that this holds for all of our boys - that something very promising is getting closer.
Kelly

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Permalink Reply by Mindy on April 27, 2009 at 6:48pm

I completely agree, Kelly. I didn't want to offend anyone, but Lindsay is getting some pretty frustrating emails, and asked me to tell everyone to tone it down a bit. I feel the desperation, too, but we do need to be careful...

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Permalink Reply by jenn on April 27, 2009 at 7:07pm

thank you mindy, i was so confused about the trial, so i asked Pat Furlong to clear things up, she did so quite well, anyone can read her response on my page if they'd like to. also, i just want to suggest that people keep the negative chatter to a minmum,this is a site for kindness, and support. we all need hope here, and need to share information in a respectful way. we also need to respect the people who are working so hard to provide treatment to our sons as soon as possible, we want to encourage the people (like mendel, and lindsay) to keep us informed on whats going on without fear of how we will react. thank you mindy for starting this discussion, i live way up here in vermont and feel out of the loop lately, its been a while since i made it to a conference, 5 kids and a family owned biusiness keeps us here...

Mindy said:

I completely agree, Kelly. I didn't want to offend anyone, but Lindsay is getting some pretty frustrating emails, and asked me to tell everyone to tone it down a bit. I feel the desperation, too, but we do need to be careful...

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