princessaurora
New Member
 Offline
Posts: 2
|
 |
« 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
|
|
|
|
|
Logged
|
|
|
|
Andy Battaglia
Administrator
Supreme Member
Offline
Location: In my heart, Maldives
Gender: 
Posts: 8656
Will thal rule you or will you rule thal?
|
|
« 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?
|
|
|
|
|
Logged
|
Andy
All we are saying is give thals a chance.
|
|
|
Parin
Junior Member
Offline
Location: USA
Gender:
Posts: 136
|
|
« Reply #182 on: November 12, 2018, 10:56:18 PM » |
|
|
|
|
|
|
Logged
|
|
|
|
Andy Battaglia
Administrator
Supreme Member
Offline
Location: In my heart, Maldives
Gender:
Posts: 8656
Will thal rule you or will you rule thal?
|
|
« Reply #183 on: November 29, 2018, 03:21:38 PM » |
|
We should see a new update after next week's presentation at ASH.
|
|
|
|
|
Logged
|
Andy
All we are saying is give thals a chance.
|
|
|
Parin
Junior Member
Offline
Location: USA
Gender:
Posts: 136
|
|
« 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 |
|
|
|
|
Logged
|
|
|
|
Andy Battaglia
Administrator
Supreme Member
Offline
Location: In my heart, Maldives
Gender:
Posts: 8656
Will thal rule you or will you rule thal?
|
|
« 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.
|
|
|
|
|
Logged
|
Andy
All we are saying is give thals a chance.
|
|
|
tahirkhan
New Member
Offline
Posts: 5
|
|
« Reply #186 on: December 04, 2018, 02:38:02 PM » |
|
Great news .
Dear Andy when can be this treatment available in market ?
|
|
|
|
|
Logged
|
|
|
|
Andy Battaglia
Administrator
Supreme Member
Offline
Location: In my heart, Maldives
Gender:
Posts: 8656
Will thal rule you or will you rule thal?
|
|
« 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.
|
|
|
|
|
Logged
|
Andy
All we are saying is give thals a chance.
|
|
|
Parin
Junior Member
Offline
Location: USA
Gender:
Posts: 136
|
|
« Reply #188 on: December 05, 2018, 01:00:06 AM » |
|
Thanks Andy
|
|
|
|
|
Logged
|
|
|
|
|
|
catchR
Active Member
Offline
Posts: 88
|
|
« Reply #190 on: April 02, 2019, 06:30:06 AM » |
|
Finally....some exciting news!!
is gene therapy from bluebird going to cost 2.12 mUSD??
|
|
|
|
« Last Edit: April 17, 2019, 12:21:38 PM by catchR »
|
Logged
|
|
|
|
Parin
Junior Member
Offline
Location: USA
Gender:
Posts: 136
|
|
« Reply #191 on: April 02, 2019, 09:08:48 PM » |
|
2.12 m USD?  |
|
|
|
|
Logged
|
|
|
|
|
Author
