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bluebird bio, a Cambridge, MA- based biotechnology company, is sponsoring the Northstar Study, investigating gene transfer as a curative option for beta-thalassemia. More information about the study, including eligibility requirements, can be found at http:///www.northstarstudy.com.
The Northstar Study is now enrolling adults who are transfusion dependent and diagnosed with a beta thalassemia major. The goal of the Northstar Study is to determine if the one-time investigational treatment, known as gene transfer, is safe, well tolerated and can decrease or eliminate the need for continued blood transfusions and iron chelation therapy.
Participation in the Northstar Study requires a commitment, and patients should expect to visit their study site often and, at times, be admitted to the hospital for various procedures, monitoring and other care. Patients may withdraw from the study at any time, for any reason. At the completion of the Northstar Study, patients will be asked to participate in a separate long-term follow-up study, as the long-term risks of gene therapy are unknown.