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No steroids ... Any other options?

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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Permalink Reply by Dave Jorgensen on July 13, 2015 at 2:18am

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.

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Permalink Reply by David on July 13, 2015 at 8:20pm

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.

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Permalink Reply by David on July 13, 2015 at 8:22pm

However, waiting for market approval is itself a risk. How long? Nobody truly knows.

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Permalink Reply by Mirtha on July 14, 2015 at 7:40am

Thank you all for your reply . I am sure I will have more questions soon . Thanks

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Permalink Reply by Dakota Ferrazzi on July 22, 2015 at 2:26pm

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.

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Permalink Reply by wainaina muiruri on July 23, 2015 at 8:42am

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.