Hello,

My little boy who just turned 8 was diagnosed with DMD a couple of months ago .
Doing all crazy research on line we find out he qualifies for the exon skipping trial for Eteplirsen .
But he needs to be on steroids for 6 months . My husband and I are not sure we want to do that , not yet. Is any parent out there giving something else other than steroids ? I want to know of supplements like Protandim and Idebenone or any other type . I read in resent news that Drisapersen and Eteplirsen have applied for FDA approval if that gets approved we may have a chance to not do the steroids ? Also is any one out there on the Eteplirsen trial ? Have you seen results ? Thank you and I am sorry for borbanding with so many questions . I hope this makes cense .
Thank you

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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.  

Could be wrong, but the steroid condition us added to trial simply to ensure the study results are not confounded by the early effect of steroid use.

I'm sure most docs will still suggest steroid use along with exon skip; but if you wait until then I doubt you'll be required to.
However, waiting for market approval is itself a risk. How long? Nobody truly knows.
Thank you all for your reply . I am sure I will have more questions soon . Thanks

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.

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