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Thalassemia Patients and Friends and thalpal © A. Battaglia 2018





54854 Posts in 5821 Topics by 6069 Members
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This is Thalassemia Patients and Friends,
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Lisa Cammilleri.
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Author Topic: Bluebird Bio Releases Data on Current Gene Therapy Trials  (Read 76984 times)
princessaurora
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« Reply #180 on: October 27, 2018, 10:54:49 AM »

Good day my daughter Aurora was recently diagnosed as being beta thalesssemia
 intermediate we live in the Caribbean as a parent I would like to know how I can get my daughter cured
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Andy Battaglia
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« Reply #181 on: November 03, 2018, 01:56:46 PM »

Right now, the only approved cure is a bone marrow transplant. This is usually only done if the patient is transfusion dependent. It should only be a few years before gene therapy will be approved and that will also definitely cure intermedia patients. Does the child take blood transfusions?
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Andy

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Parin
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« Reply #182 on: November 12, 2018, 10:56:18 PM »

bluebird bio Q3 Update

http://investor.bluebirdbio.com/static-files/37fe317c-5c01-43af-9c1d-2decb9d3800d



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Andy Battaglia
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« Reply #183 on: November 29, 2018, 03:21:38 PM »

We should see a new update after next week's presentation at ASH.
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Andy

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Parin
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« Reply #184 on: December 03, 2018, 11:42:19 PM »

First presentation of data from patients with a β0/β0 genotype and a pediatric patient treated with LentiGlobin in Phase 3 Northstar-3 study both have stopped chronic blood transfusions

10 of 11 patients with non-β0/β0 genotypes and more than three months follow-up have stopped chronic transfusions in Phase 3 Northstar-2 study

Indicators of poor red blood cell production appear corrected in exploratory analysis of bone marrow following treatment with LentiGlobin


http://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-presents-new-data-lentiglobin-gene-therapy
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Andy Battaglia
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« Reply #185 on: December 04, 2018, 09:29:07 AM »

The results are spectacular. After much negativity from some quarters about the earlier results from the non beta zero trials, we have fantastic news. Yes, only 3 patients have been started so far, but all 3 beta zero patients are transfusion free. The new protocol is working.
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Andy

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tahirkhan
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« Reply #186 on: December 04, 2018, 02:38:02 PM »

Great news .
Dear Andy when can be this treatment available in market ?
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Andy Battaglia
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« Reply #187 on: December 04, 2018, 05:49:50 PM »

bluebird bio has begun the approval process in Europe. It's only a guess, but I would hope that within two years, we will see the first approval for gene therapy for thalassemia.
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Andy

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Parin
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« Reply #188 on: December 05, 2018, 01:00:06 AM »

Thanks Andy
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