New Gene Therapy Rapidly Helps Patients With Rare Blood Disorder

June 14, 2014 2:21 PM

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Two patients who were given Bluebird Bio’s experimental gene therapy for the rare blood disorder beta-thalassemia were able to stop receiving blood transfusions within 12 days of receiving the treatment.

An earlier version of the treatment, which has allowed one beta-thalassemia patient to remain free of blood transfusions for six years, was published in the journal Nature in 2010. But that patient was not able to stop transfusions until 12 months after receiving the therapy. The new data offer hope...

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