IF I HAD ONLY HAD ONE WISH

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Offline maryo0m

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Re: IF I HAD ONLY HAD ONE WISH
« Reply #15 on: October 25, 2008, 10:12:42 PM »
Peartree Girl , yap a good treatment will do :)

sharmin,,, really? plz inform us if you had more details soon thnx  :biggrin
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Offline Sharmin

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Re: IF I HAD ONLY HAD ONE WISH
« Reply #16 on: October 25, 2008, 10:43:25 PM »
I will see if I can get access to this newsletter.  I am sure the company will post whatever results that they can 'make public' as it becomes available. 

Remember, this being the first set of trials since 2003 - when Henry Gelsinger unfortunately passed away during a gene therapy trial - the main purpose of these trials is to ensure safety.  Of course, that death was related to the nature of the disease (immune deficiency) and the nature of the treatment required to treat that particular disease - however the whole thing gave gene therapy a very bad press and delayed other gene therapy trials for years. 

Genetix Pharmaceuticals seems to have established safety - so far - and some efficacy with their first patient few patients.  Let's see how this continues to go.  Remember also the inital attempts to cure a form of blindness earlier this year, in which the dose given was minute because they were testing only for safety - and yet there were able to see significant improvement - not the full effect because the dose was very small. 

Please see the following thread:

http://www.thalassemiapatientsandfriends.com/index.php?topic=1675.0

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Gene therapy for a rare type of inherited blindness improved the vision of 4 adult patients!! 
Success was reported by two separate teams of scientists treating Leber congenital amaurosis (LCA).  As reported by the National Post in Canada. 

A common cold virus (RPE65) was used to deliver the normal version of the gene into one eye of each patient (the other eye was used as a control).  The patients went from almost complete blindness (only being able to detect hand movement) to actually reading lines on an eye chart with the treated eye. 

These patients were not expected to improve much - because they already had significant damage and were given only low doses to test for safety of the procedure.  Surprisingly, the gene therapy actually provided benefit for them. 

This is great news for patients with LCA, gene therapy and anyone with a genetic disorder.  I hope that this increases confidence in gene therapy!! 

I think that it is very important for us to understand the purpose of these studies - and watch for the ongoing results before we get either too excited or disappointed.  Frankly, I think what was said at the Singapore conference was premature and frustrating.  Gene therapy needs support and making these comments - not fully understanding the context can be harmful. 

If I come accross the CAF article I will let you all know.  Things seem positive right now form what we can all see, there is reason to hope but no one can say for sure. 

Best,

Sharmin
« Last Edit: October 25, 2008, 11:04:02 PM by sharmin »
Sharmin

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Offline Dori

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Re: IF I HAD ONLY HAD ONE WISH
« Reply #17 on: October 26, 2008, 12:39:07 PM »
September 2008/CAF newsletter What newsletter?  :dunno

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Offline nice friend

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Re: IF I HAD ONLY HAD ONE WISH
« Reply #18 on: October 26, 2008, 12:45:13 PM »
Hi Dore ,
Sharmin is talking abt the Newsletter of the Cooley's Anemia Foundation's ( CAF ), NewsLetter which issued in September 2008 .. CAF Issue's the newsletters time to time every year , which guide's toward the thalassemia its treatments and ongoing researchs ...  last CAF newsletter were published in september 2008 and sharmin is talking about to get a link of that latest newsletter ,she got that and mentioing about that in above, in her post ...
i hope it is usefull that i mentioned above if i m missing something then dont hesitate to ask that again ...

Take Care
Umair
« Last Edit: October 26, 2008, 03:37:45 PM by nice friend »
Sometimes , God breaks our spirit to save our soul.
Sometimes , He breaks our heart to make us whole.
Sometimes , He sends us pain so we can be stronger.
Sometimes , He sends us failure so we can be humble.
Sometimes , He sends us illness so we can take better care of our selves.
Sometimes , He takes everything away from us so we can learn the value of everything we have.

===========
Umair

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Offline maryo0m

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Re: IF I HAD ONLY HAD ONE WISH
« Reply #19 on: October 26, 2008, 03:23:07 PM »
Thanx nice friend and sharmin about this information I didnt know that we had a news letter  :dunno
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Offline Sharmin

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Re: IF I HAD ONLY HAD ONE WISH
« Reply #20 on: October 26, 2008, 03:45:50 PM »
you can email Cooley's Anemia Foundation (CAF) to receive the newletter:

info@cooleysanemia.org

This is the letter Bostonian refers to in an earlier post on this thread

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We received the CAF newsletter yesterday. There it talked about Gene therapy trials starting in US very soon. The newsletter also talked about the gene therapy trail going on in Paris. I forgot the name of the Doctor but the company associated with the trial is GENETIX Pharmaceuticals from cambridge, MA. The teenager who participated is doing well and continue to make some Hgb after the therapy. It did not mention if he is free from transfusion but it did mention that they already have another patient identified for theie gene therapy trial. So, may be we don't have to wait next 30 years for gene therapy to be available for our children 


this is the latest update available on the trials.
Sharmin

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Offline maryo0m

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Re: IF I HAD ONLY HAD ONE WISH
« Reply #21 on: October 27, 2008, 12:51:43 AM »
THANX ALOT  REALLY  :hugfriend
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Offline Sharmin

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Re: IF I HAD ONLY HAD ONE WISH
« Reply #22 on: October 27, 2008, 01:53:23 AM »
Maryoom,

You are very welcome  :hugfriend
Sharmin

 

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