prosensa non ambulatory exon 51 recruiting boys right now in columbus!

we just had austin's last visit in the exon 51 skipping phase one trial. we were informed that they are in need of more boys, non ambulatory for 1-4 years, to begin the trial very soon, maybe as soon as next week. this is phase one, but, it is safe to say that if FDA approves it, prosensa will be doing follow up extension trials, and the boys in phase one will not be excluded from, and most likely will be first in line to get into phase 2 and 3. this is so important, you can email me at jennmcnary@yahoo.com, or call 802-579-7471 and i will put you in touch with research trial coordinators. Dr. Flannagan asked me to spread this far and wide, so PASS IT ON so we can finish this trial and move US trials along!

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Hi Jenn,

Does this mean that Prosensa predict that their phase I may last possibly 4 years??? To then be followed by phase II and then III?
lisa, im not sure what part of my post made you think that. the only "years" i mentioned applies to how long the child has been non-ambulatory (they have to have been in a wheelchair at least one year, no more than 4 to be in the trial) this phase 1 should be wrapping up this year, or early next as long as they get all the boys enrolled. they are having trouble recruiting enough boys right now, so i am trying to find more that qualify. they expect to know about an extension study by the end of this year as well, they are waiting on the FDA to approve it.

lisa burke said:
Hi Jenn,

Does this mean that Prosensa predict that their phase I may last possibly 4 years??? To then be followed by phase II and then III?
Sorry Jenn. I just read your original post again and can see I got the wrong end of the stick. Thanks for clarifying. Serves me right for reading posts before I'm fully awake in the morning!

jenn said:
lisa, im not sure what part of my post made you think that. the only "years" i mentioned applies to how long the child has been non-ambulatory (they have to have been in a wheelchair at least one year, no more than 4 to be in the trial) this phase 1 should be wrapping up this year, or early next as long as they get all the boys enrolled. they are having trouble recruiting enough boys right now, so i am trying to find more that qualify. they expect to know about an extension study by the end of this year as well, they are waiting on the FDA to approve it.

lisa burke said:
Hi Jenn,

Does this mean that Prosensa predict that their phase I may last possibly 4 years??? To then be followed by phase II and then III?
lisa no problem, i read it over after i replied and saw i said they needed boys, non-ambulatory , 1-4 years, i can understand you thought they needed them for the trial for 1-4 yrs....anyway please pass this along if you know anyone who may be interested, it is important to further all of prosensa in the US
I hope everyone will consider signing up for this important trial. Dr. Flannagan desperately needs to get to an N number of 60 boys in order that they can then complete the extension study in which our boys will be given the DRUG!!!!!

Please please, call or email me or Jenn. Me jeanettevaughan@vntx.net jennmcnary@yahoo.com!!!!

Sure, you may think this pre-cursor study is waste of time, but the boys in this study are guaranteed a spot in the extension study (clinical trial of drug). IT is easy and they pay for everything!!!! Meals, transportation. Your boy only has to get a few sub Q injections. Easy peasy!!!!!

Please help!!!!
how do you know if your son qualifies, what deletions do that have to have? i am new to all this so i am not sure i understand .
they need a deletion correctable by skipping exon 52, there is a whole list of qualifying deletions on www.clinicaltrials.gov, or tell me the deletion and i will be happy to look it up to see if your son qualifies

donna said:
how do you know if your son qualifies, what deletions do that have to have? i am new to all this so i am not sure i understand .
I believe it is deletions correctable by skipping exon 51 and not 52. Deletion of exon 52, 50, 48-50, 49-50 are correctable by skipping exon 51.
I would love to get my son in that trial, but his stop codon is on exon 12. They are starting at exon 13. We were disappointed to see they started at exon 13. My son was so close. He has been in a wheelchair for two and a half years, so that part was good. We live in upstate NY, but Columbus would have been doable.
donna said:
how do you know if your son qualifies, what deletions do that have to have? i am new to all this so i am not sure i understand .

In addition to having the right exon for the study (see Jenn and Ana's posts), note that this is for non-ambulatory boys - not walking for 1 - 4 years.
right, sorry, didnt read my own post :) my sons have a deletion of 52, oops

Ana Vaish said:
I believe it is deletions correctable by skipping exon 51 and not 52. Deletion of exon 52, 50, 48-50, 49-50 are correctable by skipping exon 51.
This is also applicable for those with a deletion of exons 45 - 50.

Ana Vaish said:
I believe it is deletions correctable by skipping exon 51 and not 52. Deletion of exon 52, 50, 48-50, 49-50 are correctable by skipping exon 51.

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