European Commission approves bluebird bio gene therapy ZYNTEGLO listing
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Last Update: 2020-06-09
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Source: Internet
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Author: User
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blood transfusion-dependent beta-thalassemia is a serious genetic diseaseAs a result of mutations in the genes that encode "beta globulin", hemoglobin levels in patients can decrease significantly or even be losttoday, bluebird bio
(http://announced that the European Commission (EC) is in a conditional position to approve the launch of its gene therapy ZYNTEGLO, a blood transfusion-dependent beta-thalassemia for patients 12 years and olderin a number of clinicallytrial(http://, researchers found that 75 to 80 percent of patients were free from dependence on blood transfusionsAt the data cut-off, patients had not received a blood transfusion for nearly six years (56 months)This is a significant change from the frequent blood transfusions of the pastin terms of safety
researchers have observed a serious side effect that may be related to treatmentOther side effects are associated with stem cell collection and bone marrow treatment prior to transplantationThe EuropeanMedicines(http://Authority (EMA) has qualified the treatment and included it in the "Adaptive Pathway" project to speed up its review process, sources said
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