I just received a call from Paula a research nurse from Dr. Wong's staff at Cincinatti Childrens.  They are looking to start the new trials for exon skipping of 51 in hopefully September.  She stated that this was a 52 week trial... YES A YEAR! and that there is a PLACEBO!!  Every one in 3 boys will receive the placebo but the good news is that after the 52 weeks, everyone, placebo boys and Prosensa boys will be on the same drug and it will be effective after the 52 weeks.  Nicolas definitely qualifies but they haven't started the protocol since the company has changed over, and we don't have the inclusions and exclusions yet, but they want the subjects (for a lack of better term) to be ambulatory.  This is good news for Nicolas although to be on a placebo would of suck for a whole year, but at least they are freakin moving along!!!  I was told it would be one injection a week and that it could be something like a location nurse giving injection, because, you know, it would be hard for any of us in this economy, in this day, to have to relocate to another state.  So I will update you as we get new news, but this is very exciting and just a tidbit from Nicolas, he's excited because he may be able to drive, and not be wheelchair bound... My wish of course is for a CURE, but this is second best and damnwell, as a mom who feels like it's all out of my hands, I am happy to receive this news.

 

 

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My son is a Dr. Wong patient as well, and he also qualifies for this trial, but we haven't been contacted. We have definitely expressed interest in participating.

How are they deciding who to contact?
Katerina said:
What do you think about skipping other exons? We have the delition of 21 exon - it's rare. We need skipping 20 or 22 exons. Will Prosensa continue their trials for all exon or only for more freuquent? Perhaps you can ask this question for them,. I Live in Ruusia and it's difficult to me to connect with them. I'll be very pleasant if you give me any information.


No one knows if Prosensa will continue testing skipping therapies for additional exons. I'm sure it depends on the results from the initial trials, what the regulatory requirements will be for additional exons, how much they think they can make on it, and whether there'd be funding from governments or patient groups or not. I think it's too early to tell.
Hi my name is Karen and when I found out about the AVI study planned at columbus,I called and spoke with the people in
charge of the study..you can find the names and numbers through the NIH clinical studies or email me at cklf266@comcast.net and I'll look them up....They had me fax some tests {DNA and biopsey reports} then told me Lucas
was on their list and when they get the go ahead they will call all the boys on the list for interviews...the best time frame they can give right now is" later this year"....I hope this helps!!!!!

Lisa R Kelly said:
Hi my name is Lisa and I was wondering what registry you are talking about?

JoAnne Hathcoat said:
I was told Columbus is doing a trial on non-ambulatory and then they are estimating starting their exon skipping trials for amubulatory in the fall. They are still waiting on the specifics and an approved "official" start date. I am crossing my fingers for this. We are in the register there and will hopefully be included.

jenn said:
i hear through the grape vine that a non- ambulatory exon 51 trial will begin in columbus around the same tme as the ambulatory in cinci...i have one boy who would be in non ambulatory and one still running around...by next year i hope to be in both sites :)
Hi my name is Karen and I'm wondering if anyone knows if you have to be a patient of Dr Wongs to qualify for the Prosena study or if they're going to use the Duchenne connect registry??????
Did GSK and/or AVI file an IND at this point? Does anyone know?
hugs Cheryl!!

cheryl cliff said:
Me too Juan!! We'll wait along with you. hugs

JUAN PEDRO ARBULU said:
great for you guys..I´ll be waiting for 44 triasl!!!!!
best luck!
I have to say every time I check out the discussion area and see that all cap title "PROSENSA TO START SOON" it makes my heart leap. Our son has deletion 45-50, so my anticipation and excitement for phase 3 to begin can barely be contained. Who would've known I'd become such a big fan of big pharma? Go GSK/Prosensa!
Karen,

You do not have to be a patient. They like it if you are registered with Duchenne Connect, and also would be good to contact Cincinnati to see if they can put you down for this trial...

I believe I was contacted ONLY because I am on both registries for the AVI and on Prosensa. I think they wanted to know where I was going, and I told the research nurse that I'm at the point of whatever comes first, and right now, I'm hoping it's Prosensa, because it seems more advanced than AVI. We shall see!!

Karen flor said:
Hi my name is Karen and I'm wondering if anyone knows if you have to be a patient of Dr Wongs to qualify for the Prosena study or if they're going to use the Duchenne connect registry??????
thanks Michelle!!!! I'm with you..whatever comes first...I already have Lucas registered at Columbus and am trying to get
him in to see the Drs at Cinci....Thank God we have choices coming up!!!!!!!

Michelle Gonzales said:
Karen,

You do not have to be a patient. They like it if you are registered with Duchenne Connect, and also would be good to contact Cincinnati to see if they can put you down for this trial...

I believe I was contacted ONLY because I am on both registries for the AVI and on Prosensa. I think they wanted to know where I was going, and I told the research nurse that I'm at the point of whatever comes first, and right now, I'm hoping it's Prosensa, because it seems more advanced than AVI. We shall see!!

Karen flor said:
Hi my name is Karen and I'm wondering if anyone knows if you have to be a patient of Dr Wongs to qualify for the Prosena study or if they're going to use the Duchenne connect registry??????

Do anybody have information about skipping of other exons? Will Prosensa continue their trials for all exon or only for more freuquent?

 

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