EXON SKIPPING - TRIALS STARTING IN MARCH OF 2010
I just read in the on-line Quest Magazine for MDA, that Dr. Mendell's AVIPharma Exon Skipping trials are beginning in March of 2010. I signed up for Nicolas to be a part of the trials back in April and have every now and then emailed the coordinator to get updates. He did confirm that it would be taking place at that time, and right now they are getting their protocols together and should have more information in a couple weeks.
Here's my dilemma No. 1. We see Dr. Wong, and she too will be starting clinical trials for the Netherlands exon skipping drug trial (not the same as Dr. Mendell), but I have no timeframe from her of when her trials will begin. I was told last year that they would start summer of this year.... It's almost Christmas. I want something now and don't want to wait any longer.... first come first serve I guess.
My other dilemma is that my husband and I both work in Illinois and the trial is on-site in Columbus Ohio for 12 weeks, and then about 5 follow up visits, every couple weeks after that. This will of course cost me to lose my job, and in this economy, we absolutely cannot afford that. Of course my heart and thoughts are with my son and no matter what, if he is accepted into the trial, we will be there, job or no job. This is definitely more important, but I can't help but think about the future of not having a job... most likely having a home foreclose on us (we are definitely not wealthy), and living with relatives (really not close with alot of them), etc. etc. etc. Also, not having enough $$ to afford to buy Nicolas his deflazacort, and supplements, and not to mention still paying for this years visit to see Dr. Wong.
I guess I just wanted to throw my worries out there. Hopefully it's all for the best. Is anyone else up for the trials or know about Dr. Wong's???
Here's my dilemma No. 1. We see Dr. Wong, and she too will be starting clinical trials for the Netherlands exon skipping drug trial (not the same as Dr. Mendell), but I have no timeframe from her of when her trials will begin. I was told last year that they would start summer of this year.... It's almost Christmas. I want something now and don't want to wait any longer.... first come first serve I guess.
My other dilemma is that my husband and I both work in Illinois and the trial is on-site in Columbus Ohio for 12 weeks, and then about 5 follow up visits, every couple weeks after that. This will of course cost me to lose my job, and in this economy, we absolutely cannot afford that. Of course my heart and thoughts are with my son and no matter what, if he is accepted into the trial, we will be there, job or no job. This is definitely more important, but I can't help but think about the future of not having a job... most likely having a home foreclose on us (we are definitely not wealthy), and living with relatives (really not close with alot of them), etc. etc. etc. Also, not having enough $$ to afford to buy Nicolas his deflazacort, and supplements, and not to mention still paying for this years visit to see Dr. Wong.
I guess I just wanted to throw my worries out there. Hopefully it's all for the best. Is anyone else up for the trials or know about Dr. Wong's???
Replies to This Discussion
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Permalink Reply by Ofelia Marin on
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When have you found out that Dr. Wong will participate in Prosensa's trial? I think one other parent asked a month or two ago and was told that Cincinnati Childrens is not a site for exon skipping trials (?). Many parents are interested in that trial but no one heard about the location until your post. Prosensa/GSK Phase III trials are planed to start in 2010, Prosensa never said that they can start this year I think.
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Permalink Reply by Tina on
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Hi Michelle
First of all I want to say congratulations to you that there are 2 potential trials your son will be eligible to participate in next year. Things are moving and the sooner they move for your son, the sooner they move for my son as well.
I understand your concerns about your finances if you go through with the study. Would the study include your total expenses ( flights, drugs, hospitalization, medical support, etc)? Have you talked to your supervisor about any of this? I'm just curious. I can't imagine anyone giving you a hard time about taking off, after all, this is as close to a miracle as we can get here.
What about asking your community/friends/co-workers to do a fundraiser for you to help your family with the burden of your expenses while you take a family leave of absence? Just a thought. Perhaps your local church or even other organizations such as: The American Legion, The Knights of Colombus, local schools, etc , could hold a fundraiser for your family. Again, just a suggestion. The American Legion in the town where I live does them all of the time. They held a spaghetti dinner fundraiser recently and were able to send our local HS band to Florida for some sort of competition excursion.
So no matter what trial you decide to enroll your son in, you will have worked on preparing your boss for your leave and hopefully will have some cash stashed in the bank to pay the bills while you fly to Ohio with your son.
I wish you the best of luck and know your Duchenne family is standing by,waiting to hear all of the wonderful news about our boys.
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Permalink Reply by Liisa Underwood on
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If you've been with your company for at least 1 year, the family medical leave act (FMLA) protects you at your company. You many not be doing the same work, but they are required to guarantee you a job with no change in pay. You get 12 weeks per year. http://www.dol.gov/whd/fmla/index.htm
You'll have to figure out the follow up visits, but at least your good for the 12 weeks. Keep in mind that FMLA does not require the company to pay you your salary.
Good luck with the difficult decisions to be made.
Liisa
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Permalink Reply by Michelle Gonzales on
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Dr. Wong told me about Prosensa when I saw her in July for Nicolas' yearly visits....
Ofelia Marin said:When have you found out that Dr. Wong will participate in Prosensa's trial? I think one other parent asked a month or two ago and was told that Cincinnati Childrens is not a site for exon skipping trials (?). Many parents are interested in that trial but no one heard about the location until your post. Prosensa/GSK Phase III trials are planed to start in 2010, Prosensa never said that they can start this year I think.
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Tina,
I was told that there would be payment and reimbursement of expenses for hotels and travel, but it's up to the company doing the trials, and they will not have full info for a couple weeks. As for my work, it is a small, one attorney, law office. If it were a larger corporation or law firm, i might have some security, but this would put my boss in a situation without having me (her only secretary) here full time.
Thank you for the suggestion of fundraising. We may definitely have to reach out!!
Tina said:Hi Michelle
First of all I want to say congratulations to you that there are 2 potential trials your son will be eligible to participate in next year. Things are moving and the sooner they move for your son, the sooner they move for my son as well.
I understand your concerns about your finances if you go through with the study. Would the study include your total expenses ( flights, drugs, hospitalization, medical support, etc)? Have you talked to your supervisor about any of this? I'm just curious. I can't imagine anyone giving you a hard time about taking off, after all, this is as close to a miracle as we can get here.
What about asking your community/friends/co-workers to do a fundraiser for you to help your family with the burden of your expenses while you take a family leave of absence? Just a thought. Perhaps your local church or even other organizations such as: The American Legion, The Knights of Colombus, local schools, etc , could hold a fundraiser for your family. Again, just a suggestion. The American Legion in the town where I live does them all of the time. They held a spaghetti dinner fundraiser recently and were able to send our local HS band to Florida for some sort of competition excursion.
So no matter what trial you decide to enroll your son in, you will have worked on preparing your boss for your leave and hopefully will have some cash stashed in the bank to pay the bills while you fly to Ohio with your son.
I wish you the best of luck and know your Duchenne family is standing by,waiting to hear all of the wonderful news about our boys.
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Permalink Reply by jenn on
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im confused, we are also dr wongs patients, and also set to hopefully take part in the trial at nationwide childrens...but dr wong never told us about her possible trial, in fact i believe she mentioned we would have choices, prosensa or avi, in the next year but no further info. if anyone has info on where prosensa will be doing their trials id love to hear it.
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Permalink Reply by Laurie Paschal on
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I'm confused, too. We were there in August and she didn't say a word to us.
jenn said:im confused, we are also dr wongs patients, and also set to hopefully take part in the trial at nationwide childrens...but dr wong never told us about her possible trial, in fact i believe she mentioned we would have choices, prosensa or avi, in the next year but no further info. if anyone has info on where prosensa will be doing their trials id love to hear it.
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i sent her an email, she usually gets back fairly quickly, or askes andrea to answer... i will let you know what she says, also, last april when we saw her i asked about exon skipping, and she told me 51 would not work for my boys, that we need 53, we have since learned from steve wilton and others that either should do it...maybe dr wong has other opinions, i know dr brian at mass general in boston seemed to not like exon skipping at all when i asked him if that hospital may be a site for avi or prosensa... so confusing for sure!
Laurie Paschal said:I'm confused, too. We were there in August and she didn't say a word to us.
jenn said:im confused, we are also dr wongs patients, and also set to hopefully take part in the trial at nationwide childrens...but dr wong never told us about her possible trial, in fact i believe she mentioned we would have choices, prosensa or avi, in the next year but no further info. if anyone has info on where prosensa will be doing their trials id love to hear it.
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Does he give an explanation of why he thinks exon skipping will not become an effective treatment (if approved)? What does he think would be a better alternative?
jenn said:i know dr brian at mass general in boston seemed to not like exon skipping at all when i asked him if that hospital may be a site for avi or prosensa... so confusing for sure!
Laurie Paschal said:I'm confused, too. We were there in August and she didn't say a word to us.
jenn said:im confused, we are also dr wongs patients, and also set to hopefully take part in the trial at nationwide childrens...but dr wong never told us about her possible trial, in fact i believe she mentioned we would have choices, prosensa or avi, in the next year but no further info. if anyone has info on where prosensa will be doing their trials id love to hear it.
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I just received an email response from Dr. Wong who stated that trials are to begin in 2010, but no further specifics of when. As I said before, first come first serve.
jenn said:i sent her an email, she usually gets back fairly quickly, or askes andrea to answer... i will let you know what she says, also, last april when we saw her i asked about exon skipping, and she told me 51 would not work for my boys, that we need 53, we have since learned from steve wilton and others that either should do it...maybe dr wong has other opinions, i know dr brian at mass general in boston seemed to not like exon skipping at all when i asked him if that hospital may be a site for avi or prosensa... so confusing for sure!
Laurie Paschal said:I'm confused, too. We were there in August and she didn't say a word to us.
jenn said:im confused, we are also dr wongs patients, and also set to hopefully take part in the trial at nationwide childrens...but dr wong never told us about her possible trial, in fact i believe she mentioned we would have choices, prosensa or avi, in the next year but no further info. if anyone has info on where prosensa will be doing their trials id love to hear it.
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So, there will be an exon skipping trial in Cincinnati? I really wish we could get a site down here in Houston.
Michelle Gonzales said:I just received an email response from Dr. Wong who stated that trials are to begin in 2010, but no further specifics of when. As I said before, first come first serve.
jenn said:i sent her an email, she usually gets back fairly quickly, or askes andrea to answer... i will let you know what she says, also, last april when we saw her i asked about exon skipping, and she told me 51 would not work for my boys, that we need 53, we have since learned from steve wilton and others that either should do it...maybe dr wong has other opinions, i know dr brian at mass general in boston seemed to not like exon skipping at all when i asked him if that hospital may be a site for avi or prosensa... so confusing for sure!
Laurie Paschal said:I'm confused, too. We were there in August and she didn't say a word to us.
jenn said:im confused, we are also dr wongs patients, and also set to hopefully take part in the trial at nationwide childrens...but dr wong never told us about her possible trial, in fact i believe she mentioned we would have choices, prosensa or avi, in the next year but no further info. if anyone has info on where prosensa will be doing their trials id love to hear it.
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Apparently there is to be a Prosensa trial in Cincinnati. The problem is I was told this last year that it would start in summer of 2009, and to my disappointment it did not. I did not receive any further specifics from Dr. Wong, but I am not going to wait for what could be beneficial to my son and every other boy out there and fight over AVIPharm or Prosensa. Dr. Mendell's study seems to be coming along quicker and if Nicolas is accepted into his trial - I'll be there. I'd like to hear if anyone has heard from Dr. Wong on if protocols are being done, and what month of next year her trial is expected to take place.
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