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
Just saw your post (sorry I wasn't checking earlier). Sorry to hear you have joined the Duchenne group but this is a great way to interact with others who understand.
Several other people have answered about getting Deflzacort through Masters Global. My grandson was diagnosed at age 3, started taking Deflzacort at 5, now 7 1/2. We also purchase Deflazacort through Masters Global in the United Kingdom. It is very easy to get if you have a valid prescription. Need to fax the prescription to the company; go on line to set up an account; place the order and have it shipped to your house. As mentioned, the down fall is that you need to pay for it; insurance doesn't cover it because it it not FDA approved. I order it on line whenever my daughter says they need a supply. Here is a link to Master's site: https://www.masters-uk.com/medicines/home.php
My daughter chose deflazacort as it is suppose to have fewer side effects. Of course each boy's disease progression is different and their reaction to medication is different but so far we haven't seen any side effects.
If you decide to go the route of ordering deflazacort and want to discuss the process of ordering, I'd be happy to help. Email me at firstname.lastname@example.org