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.
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?????????????!
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).
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!