Discussion Forums > Working Towards a Cure

Update on Gene Therapy and Debate about Myelosuppression vs Myeloablation

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Andy Battaglia:
The second patient was started some time ago. Both patients are at home doing well and seeing a steady increase in globin gene expression and Hb level.

Sushil:
Wow... this is awesome. :thumbup

Sharmin:
This news is getting better each time - what a relief that myelosuppression is working so well.  Myeloablation is very dangerous and has many many serious side effect. 

Thanks again Andy,  I hope that we can all start planning when to sign up for the trial soon!

Sharmin

Baby_boy:


Hello to each and everyone,

My son was recently diagnosed (March 2013 - 8 month at the time) with Beta Thalassemia Major.
Since that time, I have not slept well at nights. He is the only sibling and BMT is out of the question.
Finally, Gene Therapy trials have started I must say that the updates are extremely promising. I totally believe in Dr. Sadelain and his team. May GOD bless them. From a scientific and logical point of view, Myelosuppression makes alot of sense. As Terrance's Dad, I cannot wait to have him cured.

Best regards.
Alicia & Shastri.

ANI:
I am standing at cross roads. We have almost decided to go for BMT for my child who is 8 yrs old. Seeing the good progress of Gene therapy e are confused as to to should we wait or not?
Some decision in life are bigger than the life itself.

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