Utrophin
Has anyone heard anything about Utrophin trials and what is happening with that.
I feel like the Duchenne world is moving forward which I am so happy for but what about the boys with in-frame deletions. Exon skipping will not work, PTC124 will not work, when are we going to get some good news for our boys as well. I get happy when I read about exon skipping and PTC but get upset for my nephew because not one of these breakthru's can help him.
I feel like the Duchenne world is moving forward which I am so happy for but what about the boys with in-frame deletions. Exon skipping will not work, PTC124 will not work, when are we going to get some good news for our boys as well. I get happy when I read about exon skipping and PTC but get upset for my nephew because not one of these breakthru's can help him.
Replies to This Discussion
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Permalink Reply by Keith Van Houten on
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There was some discussion on the main board in the research forum about a month ago.
http://community.parentprojectmd.org/forum/topics/updates-about-summit
Basically, the trial that Biomarin was going to do this year has been delayed. -
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