haematology
Coagulation

Gene therapy trial provides HOPE for etranacogene dezaparvovec in haemophilia B

Early data from the phase 3 HOPE-B trial in adults with severe or moderate-severe Haemophilia B supports the promise of gene therapy in protecting patients against spontaneous bleeds and eliminating the need for continuous prophylaxis. Speaking in the late-breaking session at the ASH 2020 Annual Meeting, Professor Steven Pipe said the goal of gene therapy ...

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