Excited to hear about Phase1!!! I understand that they will release more information at the end of the month but does anyone have an idea how long until they get a Phase 2 going?

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Any updates on Summit's plans?  The webinar PPMD hosted in December was so optimistic and encouraging so I'm hoping we're moving forward quickly.  Our boys don't have time to spare.

If necessary, I'll pack my bags and get my son to the UK this week!  :)


The link below is the only recent thing I have heard. I really wish they would give us more information about moving forward. I really hope they will do some trials here but would also pack our bags. My sons Doctor did say they contacted Summit when we saw him in December. If you hear or come across anything please let me know and I will do the same.



The silence is deafening.

2004 was first time I heard of utrophin upreg potential. We'll have a decade in the books before the first boy is treated. How sad is that?

Very sad.  I've been waiting to hear about some progress on this because at this point in time, it looks like the only thing out there to help my grandsons.  I feel very discouraged today.

David said:

The silence is deafening.

2004 was first time I heard of utrophin upreg potential. We'll have a decade in the books before the first boy is treated. How sad is that?

It must be the Monday, late winter blues today.  Feeling the same way today.

Since my son technically needs two exons skipped (and who knows how much dystrophin would be produced as a result), utrophin upregulation is his only hope right now.

Would be great if Summit would tell us something, even a simple as the next phase is tentatively scheduled for end of the year.  

I'm rooting for the exon-skipping therapies as hard as as anybody. But I must admit, progress at Sarepta or Prosensa actually makes me feel worse about the future for my son who is about to turn 15 because I've come to accept the fact that this industry simply will not move quickly enough to cover the entire gene in time for us.

I guess it's the worst kind of envy, and I hate that I feel this way, but it's true.

Our participation in the Eplerenone study and our intense interest in Summit is all the more intense because of this "exon envy".



Next phase to start second half of year in boys It's on summits site

Bummer it doesn't mention where the trial will take place. But it's very cool that they are essentially running 2 trials concurrently to identify valid biomarkers. That seems like it would save some time.

"The biomarker work, to be conducted side by side with our clinical development programme, will strengthen our DMD franchise and will enhance the commercial value of this asset."


" acceleration" sounds swell... let's hold them to it!

I've searched the internet for hours, emailed and called Summit, but have not found any information about the inclusion / exclusion critiera for the phase 2 trial.

I realize anything we learn now will be unofficial, but has anyone else found any hints?

If they say "ambulatory only" I'm going to scream loud enough for them to hear me in England.

I have been trying to find out more info also and have not had any luck. Did you find out when they may start recruiting or where the trial may be?


Regarding start date, all we know is H2 2013 which probably means 4th Quarter 2013.

However, the press release does indicate that more information will be released at the Muscular Dystrophy Association Scientific Conference, 21-24 April 2013, Washington DC, US. What that entails is anyone's guess.


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