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who benefits from this trial, what individual exon if you know?
They are here: http://www.avibio.com/duchenne_muscular_dystrophy.php
Exon to be skipped Drug Candidate Stage Treatable Deletions
51 AVI-4658 Clinical Development Phase 1b 45-50, 47-50, 48-50, 49-50, 50, 52
jenn said:who benefits from this trial, what individual exon if you know?
So not to sound utterly stupid here...but since Xavier has deletion of 48-52 would he qualify? or does it have to be exactly one of those groupings? We have always been under the impression, not sure why, that we would not qualify for a 51 skip.
Ofelia Marin said:They are here: http://www.avibio.com/duchenne_muscular_dystrophy.php
Exon to be skipped Drug Candidate Stage Treatable Deletions
51 AVI-4658 Clinical Development Phase 1b 45-50, 47-50, 48-50, 49-50, 50, 52
jenn said:who benefits from this trial, what individual exon if you know?
So not to sound utterly stupid here...but since Xavier has deletion of 48-52 would he qualify? or does it have to be exactly one of those groupings? We have always been under the impression, not sure why, that we would not qualify for a 51 skip.
Ofelia Marin said:They are here: http://www.avibio.com/duchenne_muscular_dystrophy.php
Exon to be skipped Drug Candidate Stage Treatable Deletions
51 AVI-4658 Clinical Development Phase 1b 45-50, 47-50, 48-50, 49-50, 50, 52
jenn said:who benefits from this trial, what individual exon if you know?
Hi Jennifer,
It looks like they have another table with more mutations and the exons to be skipped under "Information for Physicians, Scientists and Investors" on the page: http://www.avibio.com/duchenne_muscular_dystrophy.php. With a deletion of 48-52 one needs to skip 53 to make it in-frame.
Exon skipped Repaired Deletions
51 45-50, 47-50, 48-50, 49-50, 50, 52
50 51, 51-53, 51-55
45 12-44, 18-44, 44, 46-47, 46-48, 46-49, 46-51, 46-53, 46-55
53 10-52, 45-52, 46-52, 47-52, 48-52, 49-52, 50-52, 52
44 10-43, 19-43, 30-43, 35-43, 36-43, 40-43, 42-43, 45, 45-54
8 4-7, 5-7, 6-7, 3-7,
55 47-54, 48-54, 49-54, 50-54, 52-54, 54, 56, 56-62
7 2-6,8-11, 8-17, 8-43, 8-45
52 53, 53-55, 53-57, 53-59, 53-60
17 12-16, 18, 18-20, 18-22, 18-25, 18-27, 18-29, 18-33, 18-36, 18-38, 18-41, 18-44
Ofelia
Jennifer Shumsky said:So not to sound utterly stupid here...but since Xavier has deletion of 48-52 would he qualify? or does it have to be exactly one of those groupings? We have always been under the impression, not sure why, that we would not qualify for a 51 skip.
Ofelia Marin said:They are here: http://www.avibio.com/duchenne_muscular_dystrophy.php
Exon to be skipped Drug Candidate Stage Treatable Deletions
51 AVI-4658 Clinical Development Phase 1b 45-50, 47-50, 48-50, 49-50, 50, 52
jenn said:who benefits from this trial, what individual exon if you know?
do you have a phone number for lindsay at dr. mendells office? i sent an email, but i would love to follow up on monday. thank you for the info, will your son be participating?Hi Jenn,
This is new news, so it's not on the website yet. They aren't even sure of the timing yet.
It is supposed to be an efficacy study (producing results), not just a safety study.
Best,
Mindy
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