We just got confirmation about a week ago that our sweet little Owen has DMD (after 2 months of waiting for the DNA test to come back). He is 5 and has 2 big brothers (Luke age 9 and Jacob age 14). We are in the North Bay and are seeing Dr. Strober at UCSF. Our most immediate consideration is whether or not to consider enrolling Owen in the Exon 51 clinical trial and also whether or not to do steroids. We (Scott and I) are so happy that this resource exists. We have been reading a lot as we try to understand what we need to do next. Any advice from those of you who have travelled this road would be invaluable.
GSK trial says that steroid dosage has to be stable for 6 month prior to start. During the year long trial we were not allowed adjustment of the dosage due to change in weight, even though (most likely) we were on placebo. At the end the steroid dosage for us did not change for over 20 month even when we were not getting the drug. If you make a decision now you will have to stick to it till the trial gets over.
As far as enrollment is considered, its important to understand that exon skipping does not create new muscles. It just slows down the progression. I would say the earlier the better.
Thank you Tulika. How old is your son? Are you on the drug now? How long and do you notice anything?? Did you do prednisone or Deflazacort??
Glad you found our site. I am happy to send you information on steroids and other care considerations.
Feel free to email me with your mailing address- firstname.lastname@example.org or give me a call 201-944-9985.
Please also see our President and founders blog on steroids- http://community.parentprojectmd.org/profiles/blogs/steroids-a-cert...
Finally, there is a great support network in California I would love to connect you with as well.
PPMD Outreach Director
Thank you Ryan - will do!