"Mindy Hi ,
My son was resently diagnosed . I am in the USA and there are no trials for Drisapersen here but only Eteplirsen . My son would have to be on steroids for 6 months in order to quality . Can you tell me if your son finished the trial ? Is…"
Mindy's Discussions
Drisapersen Families
Started this discussion. Last reply by Mirtha Aug 24, 2015. 1 Reply 0 Favorites
In the trenches - clinical trial participation
Started this discussion. Last reply by Ofelia Marin Oct 18, 2012. 2 Replies 0 Favorites
Check out what I found...
Started this discussion. Last reply by Mindy Sep 23, 2009. 4 Replies 0 Favorites
Mindy's Page
Latest Activity
"Cialis targets PDE5 inhibition in a more targeted way (Viagra also inhibits PDE1c, and Cialis doesn't), and so the hope is that the cardiac issues seen in the Viagra trial won't apply. "
Drisapersen Families
Hello - we have gotten permission from GSK to put together an email list of parents who participated in the drisapersen study (and anyone else who just wants to know), so that when a decision comes down about the future of this study, we can receive an email from one of the orgs immediately about next steps.My son was in the Vancouver study site, and we found the day the study was halted to be quite difficult because of how hard it was to find out what was going on. This is an attempt to get…See More
"Hi Tracey - I'm very sorry about your son.
There is a trial site in Durham for the GSK study on exon skipping. If you don't want to travel as far, I'm guessing that any neurologist who is experienced enough with Duchenne to run…"
In the trenches - clinical trial participation
Hello,I'm offering the email thread below in the hopes that it might help another family who is or may participate in a clinical trial. In participation, there is not a lot that you are a parent can control, but if you think the company running the trial is making decisions that are making things harder for your son, you need to push back. Sometimes, as you'll see in the thread below, it works. Don't be afraid to stand up for your kids and what you think they need.I've replaced names of people…See More
"Their biopsy data from their original 12 boys was not double-blind, so they have not proven the surrogate marker in a blinded way as Sarepta has. They have not even released 24 week biopsy data - only the original 12 weeks from their original 12…"
"The MDA has given Sarepta 0 dollars."
"and another:
http://www.jmedicalcasereports.com/content/5/1/216"
Profile Information
- Name(s) of child(ren)/individual(s) with Duchenne:
- Aidan
- Age(s) of child(ren)/individual(s) with Duchenne:
- 4-6
- City:
- Bellevue, WA
Comment Wall (19 comments)
- At 3:42pm on November 2, 2012,
Laurie Paschal said…
It's been a week. Wanting off the DMD rollercoaster. We did finally get everything worked out with starting the OLE. We leave Monday for morning for our first trip. It's just paperwork and baseline testing (since it's been more than 90 days since Sam's last dose). Next week he gets his first dose. We have to be there Monday through Thursday for all the blood draws after. Going to be missing lots of school, which I hate, but they're being really good with it and keep Sam caught up. Did you see the FACES meeting for this Sunday was postponed?
- At 12:52pm on November 2, 2012,
Hi Mindy,
I saw your Facebook page was gone. I hope everything's okay?
Laurie
- At 11:38am on April 6, 2010,
Paul Johnson said…
- Mindy - I found this while searching some stuff... I wanted to post it to the Washington Discussion Group - but didn't want to join the group. So I wanted to forward to you and ask that you post it. Folks might find it useful (and its free to those in WA).
http://cshcn.org/care-notebook-and-care-organizer-order-form
- At 6:50pm on April 1, 2009,
jenn said…
- i promise as soon as we complete our addition i will tell you exactly how much room you need for even the most space consuming power chair...i wish someone had told me!
- At 9:34pm on March 30, 2009,
Cheri Gunvalson said…
- Hi,
I read your post about worring about your son looking like an 8 yo later on if on steroids. Our son Jacob has been steroids now for 10 years. He is 17 1/2. He walked until he was 15 1/2. When he went on growth hormone in addition to growing to 5 ft and losing wt he also lost the cushingoid sx such as the puffy face. He is also on a testoserone cream and is now in puberty...ie growing chest hair and facial hair. He looks much older. He is very glad to no longer look like a 10 yo. He is also been able to stay on deflazacort with its benifits. I don't get on this site often so feel free to email me at cgunval@gvtel.com if you have ?s.
Good luck,
Cheri
- At 1:54pm on March 6, 2009,
kerry mcnicholas said…
- 1 2 3 Magic is a behavior program for parents. Most libraries have a copy. I like it because it focuses on how to communicate with kids. It is used with kids who have disabilities too. It seems to work pretty well, but isn't as strict as time out. It doesn't always work with him, but it does stop aggressive behavior. They should sell it at amazon too. Worth a shot.
- At 7:01pm on March 5, 2009,
- We face behavior challenges often with our son. Sometimes it's anger or outbursts. It's especially bad around a transitions or changes in schedule. It's really tough to be patient. I try to use 123 magic, but sometimes it doesn't work.
- At 12:06am on March 2, 2009,
Kim Maddux said…
- Hi - I do know what you mean. The predisone causes the bloating and weight gain in most boys. The deflazacort has the same benefits with less side effects. We order it from Masters Marketing in the UK. That is why we chose it...it took us a year to make the decision. I know it's a hard one. Austin is doing great. He is 2-1/2 and talking pretty well.
fyi - Did you do IVF or PGT recently? I saw posts to you on that? I did PGT/IVF in Sept 06 and no baby. We tried again 3 months later and had healthy twins (boy/girl). Good luck.
- At 1:17am on March 1, 2009,
- Hi Mindy - Yes Alex is on deflazacort. He has been on it for 3 years. It has helped him alot. How old is you son? Have you started him on steroids. I love the pic of you 2. Take care. Kim
- At 3:40pm on February 17, 2009,
Ann Avery said…
- Mindy, Roy and I plan to go see Darius at the 1 pm time. He is just finishing his 100 days off as a MS contractor. Not being a "real" MS employee means he is not included for most of their perks, which he would enjoy. If not for his DMD he would surely be there as a FTE. Taking 100 days off after a year of full-time is really helpful for someone in his condition, but now he is ready to try to go back. It will be a totally different job as his former group has disbanded. So, he won't be bringing any matching donations.
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