Hello everyone, my son is seven years old and was recently diagnosed with DMD about a month or two ago. I would like to start him on steroids as soon as possible. His doctor has suggested entering him into a research study at Kansas Univ. hospital that will test the two possible steroids currently used (prednisone or deflazacort) to see which has a chance of slighter weight gain. Now i'm all for research but the trial seems to be a bit invasive and time consuming. I'm not sure if I want to put my son through this with this new diagnosis and himself having to cope with being different and losing strength.

Here's my problem: Deflazacort has not been approved by the FDA for the use in DMD patients in the USA yet. The only way to get this study drug is by participating in the study, even so, my son will only have a 1 and 3 chance of receiving deflazacort because there are three study or test groups and each participant is randomly selected for that group. It is a blind trial, meaning that neither I nor the investigator will know which drug my son is taking.

The question is which steroid regime do you other parents prefer/suggest and why? And what do you think about research trials.

Thanks all in advance

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Leresa,

Very sorry to hear about your son's diagnosis.  Fortunately, you've found a great organization and community for information and support.

Many of the boys in the US are getting their deflazacort from the UK without participating in a research study.  Our son has been on deflazacort since he was about your son's age.  We purchase the drug from Masters UK and have it shipped to our home in the US.  You simply need a local doctor to write you a prescription and place your order online.  Unfortunately, we haven't been able to get our insurance to cover the prescription, but fortunately, the cost is not too expensive.  Cost will depend on dosage, but I think our cost is less than $30/month.

I hope this helps!

Hi Leresa,
We also buy deflazacort from Masters in the UK and have never had a problem getting the shipment through customs. My son has been taking it for 10 years and is still walking. I did a ton of research before choosing deflazacort and felt at the time (and still do) that it was definitely the right decision for him. Donna
Which one has more effect on linear growth?
My son has been on Deflazacort for about 5 years,but he did not gain height since 1 year ago.
Is Prednisone better regarding growth?

that is a great question. i have heard nothing but positive things from families of deflazacort users.  i do bbelieve there is a difference between the two steroids.

My son has found the side effects of Deflazacort more manageable than prednisone. He's been in a power chair for 6 years. He's grown to 5'8"

David, 

Wondering if your son also took/takes growth hormone?

Thanks, Andrea

David said:

My son has found the side effects of Deflazacort more manageable than prednisone. He's been in a power chair for 6 years. He's grown to 5'8"
No growth hormones, no.

Thanks!

Andrea, I want Thuc to use growth hormones too since he has almost not gained any height for last 3 years. But his doctor said un-necessary.

How do you think? Tks/Trinh

Please check your in-box Trinh.

Thanks, Andrea.

5'8" without growth hormones and on long-term deflazacort seems very unusual...

David said:

No growth hormones, no.

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