No cure, but...: Thalassemia major is generally managed by transfusion and removal of excess iron by chelators. At some future time, she may receive a marrow transplant, or perhaps someday gene therapy will come into its own. Your sister, if she is old enough to understand, has a right to have her physicians explain everything to her; I am glad you are ready to be there for her whatever comes.
Answered 2/2/2013
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I'm so sorry: This is a genetic disease where your sister can't make normal red blood cells. She is prone to anemia which means the blood can't effectively take oxygen to the tissues. As of now, there is no cure. In the future they may be able to fix the defective gene. Right now, the only treatment is transfusions and treating any excess iron levels w chelation therapy. With these, patients can do pretty well.
Answered 4/24/2015
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