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Did The Exon Skipping 51 Drugs in The Trial Helped Non-Ambulatory Boys?

I would like to know if exon skipping 51 which are in the trial now could help non-ambulatory?

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Permalink Reply by Jason Darienzo on August 9, 2013 at 2:37pm

There are 2 Boys that became Non-Ambulatory in the Sarepta Eteplirsen trial. They are still taking the drug and pulmonary and cardiac tests are being proformed. They are showing the same levels of dystrophin as all the other boys in the trial.

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Permalink Reply by Moein on August 9, 2013 at 2:45pm

Thank you Jason,but what do you mean by your comment?Do you think that Exon Skipping did not help in spite of the new dystrophin produced by Eteplirsen?????????????!

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Permalink Reply by Jason Darienzo on August 9, 2013 at 4:27pm

Sarepta said 2 Boys that became Non-Ambulatory had the worst 6 min walk test before the trial started and were very close to going into wheelchairs and by the time the drug kicks in at 24 weeks they were Non-Ambulatory. The new dystrophin produced can only save the muscle you have, so there benefit will be in upper body strength, pulmonary function and maybe cardiac function(the drug has a harder time getting to the heart).

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Permalink Reply by David on August 29, 2013 at 11:55am

it is simply easier for the trial to exclude non- ambulatory. UC DAVIS and others are developing other outcome measures with new technology but good luck getting the trial funders to use them, the risk is too high for their shareholders.
the drug will work the same but as Jason says, it will at best preserve function not restore it.
the sad truth is that Exon skipping is a treatment for the youngsters, the older your son the less helpful it will be

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Permalink Reply by Trisha Stringham on September 1, 2013 at 1:41am

This is very sad for me to read. We waited and waited for the exon skipping trial to begin (like so many others, I am sure) than a little over a year ago my son became non-ambulatory. He missed out by a year!! It kills me everyday to see this. They need to find a way to help our non ambulatory kids!

On the plus side though my almost 6 year old nephew is in an exon skipping trial so I pray this will help him and all of the other ambulatory boys!