Deletion of exon 51

For the parents of boys with a deletion of exon 51.

Members: 19
Latest Activity: Jan 24, 2016


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Comment by Julie Hathaway on February 22, 2009 at 2:42pm
Thanks for the information. Where do you get your trial information? We would also like to be hooked up with recent news and alerts. I thought I was already?? What will help our boys with deletion 51? Help and God bless you.
Comment by Karen Barnett on February 22, 2009 at 12:34pm
Copied from another thread:

AVI will conduct the trial. It's based on the work funded by Charley's Fund ( They are working on toxicology now and plan to fill IND before the end of the year - this is my understanding from AVI's recent conference call. It will be using the new PPMOs which gave good results in mice so far. AVI did not give other details except that they hope to start it in 2010.


Karen Barnett said:
Skipping exon 50 would benefit my son. Do you know where that trial is supposed to start? Is it going to be a U.S. trial?
Comment by Karen Barnett on February 22, 2009 at 12:23pm

Skipping exon 51 will not help our boys with the deletion of exon 51. Skipping exon 50 will. This is the research we need to be watching. It is rumored to be starting clinical trials in 2010. We will see...
Comment by Julie Hathaway on January 28, 2009 at 2:12pm
Hey! I'm so glad to have found someone else who's son has an exon 51 deletion. Tell me all you know. My son, Brandon, is 6 and was diagnosed by Dr. Wong in Ohio in October of 2008. We are very new, very proactive, very strong in our faith and on a mission to find a cure. Do you know of any current studies or any similar characteristics of boys with an out of frame deletion of exon 51? Can't wait to hear from you.

Julie Hathaway

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