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Results for Gene therapy trial on Beta Thalassemia published..

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Andy Battaglia:
I would advise joining a Luspatercept trial, but with the understanding you may be given the placebo.

Luspatercept should be much more financially viable than gene therapy. That will be a big factor.

dq:
Perfect - thank you. 

I will update you if I get any new news on getting on the trial. Yep unfortunately there is the Placebo possibility :(

I'm just a little disappointed by the numbers, from what I understand only around 1.5hgb is really achievable in intermedia patients?  Or have I read that wrong and more is possible?

Thanks mate.

Sunny2701:
Sharing the below excerpt from the link

https://chicagotonight.wttw.com/2018/04/23/study-gene-therapy-blood-disorder-ends-need-transfusions

For 17 years, East Side resident Julissa Perez had to get monthly blood transfusions to treat an inherited blood disorder called thalassemia. The condition left her fatigued and resulted in jaundice, or yellowing, of her eyes. “I was tired and I would take lots of naps,” she said.

But thanks to gene therapy, Perez has not needed a blood transfusion since October 2014. “I feel great,” said Perez, who is a junior at St. Xavier University, where she's studying biology and psychology. She says it’s a “big relief” to no longer need monthly blood transfusions.

Perez was one of 22 patients to participate in gene therapy clinical trials for transfusion-dependent thalassemia. “Of the 22 patients, 15 are now transfusion independent, and what we mean by transfusion independent is they have not received a transfusion in over one year and some for nearly four years,” said Dr. Alexis Thompson, lead author of the study and head of hematology and director of the comprehensive thalassemia program at the Ann & Robert H. Lurie Children’s Hospital of Chicago.

dq:

--- Quote from: Andy on April 24, 2018, 03:02:48 PM ---I would advise joining a Luspatercept trial, but with the understanding you may be given the placebo.

Luspatercept should be much more financially viable than gene therapy. That will be a big factor.

--- End quote ---


Perfect - thank you.

I will update you if I get any new news on getting on the trial. Yep unfortunately there is the Placebo possibility

I'm just a little disappointed by the numbers, from what I understand only around 1.5hgb is really achievable in intermedia patients?  Or have I read that wrong and more is possible?

Thanks mate.

Andy Battaglia:
dq, I don't think we can draw any conclusions about the long term effect of Luspatercept until the phase 3 trial results are reported. I do know that some patients in the trials are doing very well.

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