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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Hi Amit


I would suggest that you e-mail PPMD and ask for a copy of the Connect 2011 Annual Conference program.  It contains a summary of each research project that was presented.  It may be on-line, I just don't know where to direct you.


Best wishes,


The majority of the presentations can be found at this link

Just was listening to the MP3 lecture about Pro 044 by Giles Campion,he did mention a fact that for boys w/spantaneous mutation,this pose a problem w/exon skiping.

If anybody have an explanation what that mean?Please Reply.

Thank you.

Thanks Jon. I suppose you can't make any comment on your funding status?

I found no new information in the conference summaries on these topics. Plenty of vague statements about "ongoing analysis" but no substance in any area other than exon-skipping.

The "announcement" by Shire re:ACE-031 was no different than what they have on their web site.




No mention /update for Ataluren extension trial neither..
I also found that nothing new was reported and no timelines. In a couple of cases, for example VBP15 replacement to steroid Hoffman's company works on, I found that the timeline announced by Eric Hoffman more than a year ago (during the Action Duchenne conference and other instances) was very far off. That drug is not even in pre-IND stage. When it gets to pre-IND will take more than one year to get to trials, but for now the drug is not in pre-IND. Very disappointing to say the least.

I also noticed that all statements regarding utrophin upreg treatments under Project Catalyst when from "end of this year" to "end of 2012."

The feeling of powerlessness is overwhelming. Waiting a decade now, and never has a any company lived up to any timeline they've every put out.

I am overjoyed that a handful of boys in the magic exon-51 range are doing better in trials. I am also hopping mad that a community built on the understanding of "rare disease" seems to be most aggressively pursuing therapies that exclude 80% of those affected.

From outsiders point of view, this conference reinforced my fears that those outside of the magic exon ranges are out in the cold.


David ,i think as parents we all feel like that .Days i am so optimistic and days where i feel just totally overwhelmed trying to fight this for my son!! There is so much hope with all these potential treatments but it is taking time,and time is muscle....arrgh! I get so frustrated about Ataluren i cannot even begin to explain!!

I was under the impression that Summit was expected to know something about funding this month (from PPMD). It would be tremendously helpful if SOME information came on that front. Perhaps Jon Tinsley can comment on what the decision times for funding from other possible sources are?

The silence and secrecy add to the frustration.

The first thing I figured out in my 3.5 years in this community is to take everything the scientists, foundation leaders etc. say about any of these compounds tested with a grain of salt. There are some very few exceptions, people who honestly tell the truth, but the majority just think that telling the truth about these compounds being very far from trials let alone approvals would make parents feel bad and make them not interested in fundraising to support the work.

For me is the exact opposite...if you lie that your compound will be in trials within 6-12 months even though you are not even in the pre-IND phase, then you lost credibility and nothing you say from then on makes me want to support you. Of course they do not want to truthfully tell us that the majority of these possible treatments will not be here in time for our sons...who would continue to support them if they say that... I am not interested in making my son a scientific experiment so that the next generation can have a treatment.

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.

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