My understanding is that exon skipping works better with steroids, so we as a community are stuck with them for now. There is possible replacement (VBP15), but approval is far off. Steroids are a big debate for some and the decision is yours, but I think the preponderance of data shows the benefits outweigh the issues. Since none of the drugs are approved you'll have to wait or enter a clinical trial. You could look into Akashi's HT-100. The results look good and they accept patients who are not on steroids, but keep in mind if you put your son on steroids you'd have to be on a stable dose for a year to be in the study.
i have been on steroids since i was 3 years old.. I'm now 20 years old and still walking… putting your son on steroids and hopefully getting the eteplirsen could allow him to live a normal happy life.
Hello Dakota.I trust you are well.Have you ever had temperament and weight problems with the steroids.I had my son on them but had to stop after two months because his weight shot up rapidly and he had a really bad temper.What can one do to mitigate the effects of steroids?Thanks and have a good day.