Gene therapy Valoctocogene Roxaparvovec For Severe Haemophilia A: Long-term Data Again Validates Effectiveness and Safety
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Last Update: 2020-06-24
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Source: Internet
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Author: User
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BioMarin Pharmaceuticals recently announced that the gene therapy Valoctocogene Roxaparvovec for the treatment of severe haemophilia A's Open Label I/II study has yielded new results to be announced at the World Hemophilia Alliance (WFH) Summit from June 14 to 19, 2020four-year update to the 6e13 vg/ kg group and the three-year update to the 4e13 vg/ kg group showthat that all subjects in both queues have not received factor VIII treatment since receiving a single dose of valoctogene roxaparvovecIn both queues, the cumulative average annual bleeding rate (ABR) is less than 1 and below the pre-treatment baseline levelThe average ABR for the fourth year of the 6e13 vg / kg queue is 1.3, while the average ABR for the third year of the 4e13 vg / kg queue is 0.5In the past year, 85% (6/7) of patients in the 6e13 vg/kg queue and 83% (5/6) in the 4e13 vg/kg queue did not experience spontaneous bleeding"These additional data are an important step in gene therapy for the treatment of severe haemophilia A," said DrJohn Pasi, lead researcher on thestudyThis is a good way for this destructive diseaseEach year's data increases our understanding of safety and effectiveness."
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