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our two boys have deletion of 52, although clinically they couldnt be more different, max is almost 8, he runs, jumps and plays, he seems to get "better"... austin is almost 11, and has used a scooter for 4 years, he just got his first power chair. we have been contacted by nationwide childrens in columbus, for pre enrollment data for the avi trial of exon51 skipping that will possibly begin in march.has anyone else shown interest in that trial? we hope that both boys will get in, we will take any improvement we can get for austin.
Rhiannon - how do you know you're out of frame at the MRNA level?
My son has an in-frame deletion and is progressing as DMD also.
Can they test the MRNA somehow?
I guess I don't understand how you get the mRNA from a biopsy. I thought a biopsy tells you if you have dystrophin present. Maybe I don't understand what mRNA is properly. I understood it to be a copy of the DNA that is made and used for creation of the protein.
Can you explain it in simple terms? Send me a private message if you want to continue this off thread.
Thanks, Keith
I guess I don't understand how you get the mRNA from a biopsy. I thought a biopsy tells you if you have dystrophin present. Maybe I don't understand what mRNA is properly. I understood it to be a copy of the DNA that is made and used for creation of the protein.
Can you explain it in simple terms? Send me a private message if you want to continue this off thread.
Thanks, Keith
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