Summit's SMT C1100 gives more hope in that it has the potential to treat all patients with DMD despite the underlying causes of the condititon. How long before it gets into the market only God knows. Wishing the team well.

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You are right at this moment SMT C1100 seems to be the best hope for all of us. Exon skipping is very mutation specific and is also going through ups and downs and don't know when it will be available. All other trial seems to be for slowing down the progress of the disease and not for cure. Correct me if I am wrong.

Rahul, I don't think SMT will cure DMD unless it's given at a very early age, but it hopes to stop the disease and see some regeneration, but not a cure as such, although you're right it applies to the whole DMD population and not gene specific. 

Steven I was under the impression that SMT will produce ultropin which will act as a substitute for dystrophin. Is it like other drugs under trial which are trying to slow the progression of the disease?


Rahul, yes that's right,

I think the difference  is in the language in that a cure suggests someone would be free of disease and recovers from it, while I think most or all the drugs in trial are trying to halt the disease and provide some improved function but that's not the same as a cure.  

I personally have little faith in summit. They give rosy press releases and miss every date it seems. They move as if in cement
They want you to think that is normal but look at Akashi/Halo and HT-100 and you'll see how fast a trial can move if the sponsor is actually motivated.
I'm no expert, just opinionated :)

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