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Thalassemia Patients and Friends and thalpal © A. Battaglia 2017





54174 Posts in 5694 Topics by 5774 Members
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We love you, Lisa.
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Author Topic: New Gene Therapy Trial Results  (Read 9020 times)
Pratik
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« on: December 15, 2014, 11:27:38 PM »

Hey friends,

I have been away for a while but I couldn't help but to step in and announce what I read yesterday in CAF's (Cooley's Anemia Foundation) newsletter.

There has been some exciting and heart soothing results in regards to the gene therapy trials currently undergoing at Sloan Kettering.

Here is what it said:



This has made me very optimistic and let us hope that we have a fully functional treatment available soon!

Best,

-P
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Himanshu Kumar
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Aditi


« Reply #1 on: January 01, 2015, 11:10:48 AM »

Read this on my facebook wall. This is a story of 33 year old patient who successfully received bone marrow transplant.

http://josephinebila.com/2014/12/bone-marrow-transplant/
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Regular transfusion, Keeping pre transfusion HB above 10 and Iron Chelation are the three mantras for thals to stay healthy.
Himanshu Kumar
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Aditi


« Reply #2 on: January 01, 2015, 11:25:38 AM »

Another story on Gene Replacement Therapy

http://wgntv.com/2014/12/30/gene-replacement-could-replace-need-for-life-long-blood-transfusions/
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Regular transfusion, Keeping pre transfusion HB above 10 and Iron Chelation are the three mantras for thals to stay healthy.
Andy
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« Reply #3 on: January 01, 2015, 12:28:03 PM »

Nice to finally see a report from an actual gene therapy patient. Wishing the best for Wanda.

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Andy

All we are saying is give thals a chance.
Andy
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« Reply #4 on: January 01, 2015, 12:29:56 PM »

The person who received the BMT and was interviewed by Josephine is David Levy, a long time member of thalpal and founder of the thalassemia support group on Facebook, which I also administer. It is so exciting to see an older patient cured.
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Andy

All we are saying is give thals a chance.
Himanshu Kumar
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Aditi


« Reply #5 on: January 02, 2015, 11:01:53 AM »

Absolutely, it is so heartening to see older patients getting cured. Gives hope to many patients. Great to hear about successful cases on Gene Therapy. If the early results are so encouraging, why aren't they recruiting many more patients. I sincerely hope the treatment is made available across the world at a low cost.
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Regular transfusion, Keeping pre transfusion HB above 10 and Iron Chelation are the three mantras for thals to stay healthy.
jay
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« Reply #6 on: January 02, 2015, 05:16:51 PM »

Himanshu,
We have clinical trials -> FDA regulatory process ->Price skimming
and then we can see it near to us ... loooong way to come to gene therapy.
Regarding recruiting more patients: clinical trials are normally the most costly affair in the process.
By the way Himanshu not to confuse with David Levy's BMT with gene therapy, he gone through a unrelated full match BMT, not gene therapy.
Regarding low price BMT, assuming you are from India let me tell you if you have complete HLA related match (not just siblings it is also highly possible to find a related donor in cousins) then it could be done in five lac (RS 5,00,000).
Regarding BMT in older patients it has been there for a while we are excited here as he is one of us we talk.
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Pratik
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« Reply #7 on: January 06, 2015, 01:28:18 PM »

I just came to post this and saw you posted it. Great!

I just received newsletter from CAF which contained link to it and thought on sharing here.

Yes, its amazing to see the testaments of new treatment. Couldn't be more excited for all of us!

Best,

-P
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Parin
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« Reply #8 on: January 07, 2015, 01:48:09 AM »

Thank you for sharing the updates...its makes happy everyone.
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nice friend
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« Reply #9 on: January 23, 2015, 06:36:20 AM »

its a  ray of hope for older thals ... once again feeling hopefull for a bright future ... 
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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.

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Umair
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Little A


« Reply #10 on: January 25, 2015, 05:34:34 PM »

You have every reason to be hopeful Umair.  You have come a long way with chelation etc.  Continue to take good care of yourself so that you can take advantage of the cure when it becomes widely available.

Sharmin
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Sharmin
nice friend
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If I Can, Why Not You??... If I Can U TOO !!!...


« Reply #11 on: January 30, 2015, 05:02:13 AM »

sharmin ,
i m trying my level's best to keep everything in normal range and fine and its all wat i can do ...
but sometimes you dont know when n why/how something wrong happen's  to you and takes you off track again....   :/

anyways still am not gonna loose hope ,, i will keep trying my levels best to keep everything in normal ranges ..
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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
Andy
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« Reply #12 on: January 30, 2015, 11:49:44 AM »

  Umair.
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Andy

All we are saying is give thals a chance.
nice friend
Thalassemia Major
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Location: Faisalabad, Pakistan.

Gender: Male
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If I Can, Why Not You??... If I Can U TOO !!!...


« Reply #13 on: January 31, 2015, 05:28:08 AM »

thanxx Andy u always been a big source of motivation and encrougement fo me  .. .
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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
Pratik
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« Reply #14 on: February 05, 2015, 12:52:03 AM »

Umair,

It is so good to see you after long! I feel members should keep turning in at least every few months so we can know what's going on with everyone's lives and how well they are doing.

Keep it up, my friend!

Best,

-P
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