Duke scientists have developed a way to change the existing mutated gene responsible for the disorder into a normally functioning gene.



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Would this help all dmd patients?

Hello Jason

I hope your week is going well for you and your loved ones.Now,this info sounds so good its almost unbelievable.And the article says this approach can actually treat about sixty per cent of dmd cases.This is the kind of news that a dmd family yearns to come awake to every dawn that breaks.But there is a but;time.May be by the time this concept is up for public consumption,lots of other cures already in advanced stages of development will already be in use.But this news is just the greatest by any means.

"..this info sounds so good its almost unbelievable.." 

That's what went through my mind. When the article states.."..their approach could be safer and more stable than current methods.." &  "Our approach actually repairs the faulty gene, which is a lot simpler,..", you can't help but feel optimistic.

".. there is a but;time"

As always, the time scale in these things is vital. Please god, it's simple, safe, successful & available *QUICKLY*.

Looks like they hav taken care of the simple,safe and successful aspects of the product;the quickly is the critical now for us.Let us pray that this comes fast enough for all of us in the dmd community.Amen.

Time is always an issue for DMD patients and this new approach will take years to get to clinical trial.

However, the current DMD pipeline can bridge the gap to this new more permanent solution.


Looking forward to the next update. Thanks for the other good new and another piece of hope. If this was it....

what can we do to speed this up?

I've just communicated with Pr. Jacques Tremblay who is part of the team, he said the technique would help all dmd patients, whatever their mutation is.

That's great to hear!

Parlava di 60 per cento ,perché si riferiva ad un esperimento sull'esone 51 che riguarda il sessanta per cento dei malati di Duchenne,ma il sistema servirebbe il cento per cento dei casi!!


Hello Galia

That is real good news.I know this is still pretty early to ask,but kindly can you check with the good doctor the approximate duration of time this product might take to enter into the market.Thank you.

the doctor said it is in pre clinical stage. Please see the doc. he sent me:


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