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PTC124 - New drug may cure genetic diseases

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Parin:
http://www.sciencentral.com/articles/view.php3?article_id=218392919

christian:
hi to every one, my sons hematologists recommends him spleenectomy, do u have any relative or do  urself submit for spleenectomy? what is the result? does it lessen the trnsfusion? my son is being trnsfused every three months..he was diagnosed last year for beta thalassemia major

Manal:
Hi Christian and welcome to the site

I guess  that your son is a thalassemia intermedia since he is now 11 years and he was only diagnoised last year. What is his Hb level ??

Manal

Sharmin:
Wow, the drug sounds amazing!
Andy, what are your thoughts?

§ãJ¡Ð ساجد:
O.k is Thal. a "nonsense" mutation? :huh

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