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Male1: The life expectancy we get with that of diagnosis?
Male2: Hard to say, but most with Desferal, patients with Thalassemia have lived into their 30 or 40s and beyond, if well chelated. It is our expectation that with EXJADE, actually that will go even further. The other thing that should be noted about Thalassemia is that Thalassemia is not only treatable, but it is potentially curable because bone marrow transplant has been proven and is an established way to essentially cure people with Thalassemia.
Male1: What percent of your case with Thalassemia of the true disease end up with bone marrow transplants?
Male2: Until recently, the vast majority of patients who could be transplanted were patients who actually had a sibling, there is a brother or a sister who is in perfect match or an acceptable match for bone marrow transplant. The people expecting transplantation were not in favor of using what is called unrelated donors--those are donors that were obtained from the general population through a registry because of the risk benefits. Transplantation though has improved over the last two decades and recently, there is that thought that unrelated donor could be used for patients with Thalassemia which would open up the possibility of more patients with Thalassemia being transplanted. It is a little hard to tell you what exactly the statistics are because it depends on the size of the family each time there is a sibling, there is a chance in four that that sibling is a full match for that particular patient, so it depends on the size of the family.
Male1: So it is a disease that can be managed.
Male2: It is a disease essentially that can be managed and it is a disease that has a potential for cure. In addition, there are all kinds of studies that have been going on for a while that are even thinking about the possibility of so-called gene therapy, where essentially, a new gene will be given to the patient who has this genetic problem in the hope that that would be treated. And I think that during the infancy, and I think we have a way to go into that area, but a patient with Thalassemia who is well transfused and well chelated has at this point, in our mind, a life expectancy that is very good and has the potential for cure through a bone marrow transplant if the circumstances are right.
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