So this week, one of our biggest hopes came true.   About a year ago, I left my job to take a consulting position and we moved to British Columbia.  We weren't particularly happy with the services we were receiving in Saskatchewan and decided if we could, we'd move to be closer to one of the Children's hospitals in Canada.  We didn't want to move East to be near Toronto, so BC was the only other choice.  There certainly were some stressful times during the move and since, and it wasn't easy moving away from both my and my wife's family, but this week we got some great news.

We'll be going in to have both our boys screened in for the Pfizer Myostatin inhibitor trials.  The timing couldn't be closer though.  Hayden, our oldest, will turn 10 in November and only boys under 10 can be included.  In talking with the coordinator, we learned that they have to be 9 at the time of randomization, which should happen sometime in October. 

This is one of a few trials I personally was really excited about.  The other two being exon skipping  which is likely at least a few years away for skipping exon 44 (or 46) and utrophin upregulation.  I hope, for all of us, that these all go well and that in the relatively near future, these treatments can be combined and give our children a drastically different outlook than the one they have now.

Views: 169


You need to be a member of PPMD Community to add comments!

Join PPMD Community

Comment by Andrea Cleary on August 15, 2015 at 3:19pm

In the nick of time. What a roller-coaster ride. Best of luck and science.

Comment by David on August 15, 2015 at 2:52pm
Good luck!!! We also wait anxiously for Epicatichen trial that takes boys 18 and under, and my son just turned 17.
On the good side, my finger naiks stay very trim:)

Need help using this community site? Visit Ning's Help Page.



© 2022   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service