princessaurora
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« Reply #180 on: October 27, 2018, 03:54:49 PM » |
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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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Will thal rule you or will you rule thal?
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« Reply #181 on: November 03, 2018, 06:56:46 PM » |
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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
All we are saying is give thals a chance.
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Parin
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« Reply #182 on: November 13, 2018, 04:56:18 AM » |
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Andy Battaglia
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« Reply #183 on: November 29, 2018, 09:21:38 PM » |
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We should see a new update after next week's presentation at ASH.
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Andy
All we are saying is give thals a chance.
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Parin
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« Reply #184 on: December 04, 2018, 05:42:19 AM » |
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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, 03:29:07 PM » |
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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
All we are saying is give thals a chance.
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tahirkhan
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« Reply #186 on: December 04, 2018, 08:38:02 PM » |
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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, 11:49:50 PM » |
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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
All we are saying is give thals a chance.
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Parin
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« Reply #188 on: December 05, 2018, 07:00:06 AM » |
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Thanks Andy
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catchR
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« Reply #190 on: April 02, 2019, 11:30:06 AM » |
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Finally....some exciting news!!
is gene therapy from bluebird going to cost 2.12 mUSD??
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« Last Edit: April 17, 2019, 05:21:38 PM by catchR »
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Parin
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« Reply #191 on: April 03, 2019, 02:08:48 AM » |
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2.12 m USD?  |
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