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Results for Gene therapy trial on Beta Thalassemia published..

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Sunny2701:
https://www.npr.org/sections/health-shots/2018/04/18/602914728/gene-therapy-for-inherited-blood-disorder-reduced-transfusions


Results of a study published Wednesday show that 15 of 22 patients with beta-thalassemia who got gene therapy were able to stop or sharply reduce the regular blood transfusions they had needed to alleviate their life-threatening anemia. There were no serious side effects.

"We're extraordinarily excited about these early results," says Alexis Thompson, a professor of pediatrics at the Northwestern University's Feinberg School of Medicine, who helped with the study released Wednesday.

"For the first time ever, we have a treatment that we might offer to all our patients," says Mark Walters of the University of California, San Francisco, who also helped conduct the study.

Andy Battaglia:

--- Quote ---Bluebird Bio of Cambridge, Mass., plans to seek approval of the treatment in Europe by the end of the year, a spokeswoman said in an email.
--- End quote ---

Andy Battaglia:
I have confirmed with bluebird that approval is being sought this year in Europe.

Sunny2701:
That's wonderful news Andy, thanks for reaching out to them

dq:

--- Quote from: Andy on April 20, 2018, 10:24:26 PM ---I have confirmed with bluebird that approval is being sought this year in Europe.

--- End quote ---

Andy, the Luspatercept trials have opened their doors for Thalassaemia Intermedia and from what I understand the trial is running for 46 weeks at minimum.

Two different treatments entirely but;
Would you recommend holding of starting the trial if one has the option of joining it in the hope that Lentiglobin gets approval sooner and is a better treatment option?
Or would you recommend going on the Luspatercept trial if at all possible?

Thanks pal.

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