Five months ago I found this forum and hoped that it would be a source of information about Duchenne. Unfortunately I found that participation in this once active forum has significantly decreased. Fortunately a member tipped me off that everyone had moved to Facebook group. I received an email notification of an response to an old post today and thought it would be good to post links to some of the active Facebook groups;Duchenne Under 10 - ~1,500 members. An active group discussing…See More
I was wondering if your son is still using tamoxifen and if the situation is still stable or better. Is your son also using prednisolone or Deflazacort? And are there any side effects with tamoxifen? Can you also tell me when your son is…"
I was wondering if your son is still using tamoxifen, if so, how is he doing? I also want to know how you started it. Is a doctor checking your son, or are you doing it all by yourself.
"Amazing job with this! Certainly will look into doing this, really helpful that it's all ready to go! Lol! I will contact you guys at PPMD when I am ready. Thank you Diana for fighting for our boys and all of you at PPMD!
"Will each trial run the same test and would that mean I would be excluded from all studies?
All of these first studies are using AAV and will be running the same antibody titer testing.
While it is true that all the groups will be doing the…"
In January of 2014 we held our inaugural PAAC Leadership Workshop in Washington,DC. The day included engagement with federal policy makers, congressional staff, and research leaders. At the end of the day, a long-time colleague of mine from the FDA who had spent the entire day with us pulled me aside and said, “Wow. These men…See More
"Thanks a lot for all your lead in this DMD world. It is helping our kids and sure coming generations. Micro Dystrophin transfer research is life changing for our boys and girl. we really need these type of drugs/research more. we should not stop…"
"UPDATE: Ok, it was seven years ago when I last posted on this thread about my nephew, Byrum Bittel. The kid who was not supposed to make it to 16 years old is now 22. Been in college and now advancing to medical records management."