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If we can envision treating patients as if the patients were their own donors, they are not exposed to some of the risks that come from transplants when you're using someone else's stem cells, relatively speaking, this procedure was well-tolerated by the patients, with no unexpected toxicities related to gene therapy.
Found on CNN 6 years ago
There were no novel side effects identified that related either to the LentiGlobin vector or the gene therapy procedure itself, we clearly want to watch for a much more extended period of time to be sure that there are no additional safety concerns.
Found on CNN 6 years ago
The things that I would note, however, are that one needs to evaluate the overall cost of patients who undergo sibling transplants and see if it is comparable, when we look at what the alternative might be -- having a stem cell transplant of any kind -- that is one number that has to be borne in mind. The second is looking at what the costs are for annual care for individuals with thalassemia.
Found on CNN 6 years ago
The side effects that were seen in these two clinical trials are consistent with what we expect from a transplant of any kind that uses chemotherapy.
Found on CNN 6 years ago
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