I was not able to go to the conference and am wondering if anyone who attended could provide what was said about the following:


1. ACE31 trial - when is it likely to resume?

2. Summit SMTC1100 trial - are they expecting funding and starting this year?

3. VBP15 trial - this year?

4. Any other positive news



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Completely agree! We move from conference to conference hoping for some sort of news when there isn't much at all. We'll definitely hear about WMS soon, then Action Duchenne's conference and so on, just to hear the same presentations and the always extended timelines.

David said:

In a way it seems to me these conferences can discourage honest communication from the trial sponsors. All year long we hear "can't say much now, but attend PPMD Conference and learn more!" and then when those conferences come around, we get the same old information.

I'll bet you $100 that when I see my clinician next month he will say "Well, World Muscle Society conference is September, I'm sure we'll learn more then". Yea, sure.

I remember the AD Conference in London last year!!! Some things that stuck in my mind...1. AVI wouldn't even put the presentation on the net as there were parents in the room who were almost physically sick, or bursting with anger due to the way their sons had being treated on trials(with no option of continuing, even though the boys had improved/made dystrophin) . 2. A break out session with EMEA, who said to the audience that they  say to companies.."show us something that works, and we will allow it on the market, we are here to help"... Then a person who works with a pharma company commented, "well, it would be helpful if you could reply to a dossier, instead of the dossier being left on a desk for over a month!"... Great!!!!  3. Spoke with one man who has worked on exon skipping for years....his attitude was, as far as I could see is...get the drugs made for these boys..quickly!!.. get 51 skipping on the market and follow on with everything else ASAP!!!....... It just seems to me that everyone is not pulling in the same direction? can be frustrating and depressing at times!!! 
I just read an article about AVI4658 update.

Promising results published today in the Lancet on Exon Skipping to treat Duchenne Muscular Dystrophy tinged with disappointment as extension study is not undertaken


Can someone explain 1. Is Exon 51 skipping drug AVI4658 being introduced in market soon. 2. Is it being introduced in market without an extension study?

Can somebody in UK approach UCL and ask for latest updates.

There will be a 24-week study starting next month at Nationwide Children's in Columbus Ohio testing higher doses (30, 50 mg/kg). If successful, there will be longer placebo controlled pivotal studies starting end of next year the earliest. You can listen their presentation from PPMD conference for details.

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