PROSENSA TO START SOON
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.
Replies to This Discussion
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Permalink Reply by Melanie on
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I know how exciting such news can be. I hope things work out for you and Nicolas, and that the trial results in the news we all want to hear. :)
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Permalink Reply by JoAnne Powell on
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This is great news! I wish you all the best of luck! Wow! A YEAR! I have been waiting to hear from Dr Mendell's team in Columbus and have my fingers crossed we hear soon as well! I hope this exon skipping works as well as they are hoping it will!!! Very exciting news...thanks for posting!!!
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Permalink Reply by Tamara Walters on
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Same thing with my son and the PTC trials. A year, and maybe placebo...but at least it is forward motion ! And whatever happens, it is not for nothing. They have something to gain in all research.
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Permalink Reply by Ofelia Marin on
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What do you mean by PTC trials? Is there another PTC trial starting?
Tamara Walters said:Same thing with my son and the PTC trials. A year, and maybe placebo...but at least it is forward motion ! And whatever happens, it is not for nothing. They have something to gain in all research.
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Permalink Reply by JUAN PEDRO ARBULU on
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great for you guys..I´ll be waiting for 44 triasl!!!!!
best luck!
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Permalink Reply by cheryl cliff on
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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!
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Permalink Reply by Roderick Scott on
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Has the Prosensa trial been confirmed?
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Permalink Reply by Darcy Tumminello on
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Is there a list of exons that they are currently working on skipping in order? Any word on exon 50 or 56? My son has deletion 51-55.
Thanks
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here is the list
http://www.prosensa.eu/technology-and-products/Pipeline/index.php
Darcy Tumminello said:
http://www.prosensa.eu/technology-and-products/Pipeline/index.phpIs there a list of exons that they are currently working on skipping in order? Any word on exon 50 or 56? My son has deletion 51-55.
Thanks
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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 :)
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Permalink Reply by Katerina on
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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.
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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 :)
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