Hi Ana,

 

there are several options:

www.miracleflights.org

or through continental - email malia reed at careforce@embarqmail.com (this is how you request a flight through continental's program called Careforce)

or private planes:  maybe angel flight..if close enough or a group called Wings of Hope (google either one of these).  I have flown all except Angel flight. Wings of hope is a small private plane.  all of these will fly one patient and one parent...

Good luck!

Thank you so much! My son is too young to participate but I love to see things progressing!

Good. Just wanted to make sure that you know. Did she say when they expect the trial to start? Any other info about how exon skipping works so far? Does she think that this will be approved in 2-3 years?

Hi Ana,

I hope all is well.
Please take a look at the latest discussion about the exon skipping trial. Someone posted that Dr. Wong told her that they are a site for the Prosensa trial. I think you emailed her a couple of months ago to ask, but I remember she said no at the time (??). So you might want to ask Michelle about it.

Ofelia

About the comment under mine, in Annemieka's last paper or it might have been tonight, she talks about there being a few different variables between the studies and so I agree they could be very similar in outcome until all conditions are made identical.

Hi Ana, I'm sorry I didn't check with DMD community while the chat was ongoing. I didn't think to. When you say you couldn't connect, have any attempts to open the conference room URL worked for you?
http://www.hotconference.com/conference,49190604
What web browser are you running? Internet Explorer? I was unable to record the chat as well, I was booted a couple of times from the room and that lost the recordings I had initiated. Other than that we heard Annemieka well and saw her presentation and her face. I don't think the dozen others got booted out as much as I did. I am only learning and will make sure I check this next time. We had a practice chat last two nights leading up to tonight. Let me know if the room doesn't work when you try it in next couple of days and your OS and I'll ask the software people?

Hi Ana,

I do not know if it will be safety only. My impression is that it will not be safety ONLY since the AVI ceo says that they plan to use the dose levels found working in the UK trial.

People say that AVI's chemistry is "better" than Prosensa's but if you look at the comparison b/w % dystrophin in the intramuscular trials they seem to be performing the same. What's important is what % is obtained in the systemic trials. Prosensa does not say how much dystrophin they obtained and AVI will let us know end of year...then we can have a better idea if one is better than the other.

Got to run. Talk to u soon.

Thanks Ana: I did fin out some information.
I just got this email in from the Ohio folks where the AVI trial is slated to take place.
Hello,

You contacted Lindsay Arnott who used to work in our institution, back in April and she had a chart open for your child. We have his name, date of birth and mutation and he is included in our list of potential candidates.
However, we are not ready to start the trial yet. The sponsor, AVI, will give his “go”, hopefully in a few months, and we will start enrolling patients. We will contact at this time to proceed to enrollment and screening. We would be very happy to have Liam in the trial and we will make sure to contact you back.
In the meantime, if you have questions about the trial, please feel free to email me.
Thank you very much.
Best regards,

Laurence

Laurence Viollet, PhD
Clinical Research Coordinator
Team Project Leader
Center for Gene Therapy
The Research Institute at Nationwide Children's Hospital
700 Children's Drive
Columbus, OH 43205
Phone: (614) 355-2695
Fax: (614) 355-5247
Laurence.Viollet@nationwidechildrens.org

What's this EMEA/FDA meeting in london this weekend? I know nothing about it - just the conference in London in October. Do you have a link???
Thanks