Biomarin presents new five-year data for hemophilia gene therapy
Gene therapy valoctocogene roxaparvovec had a turbulent start, but Biomarin has now released five-year data for the treatment, which is crucial to getting FDA approval.
BY ELIZABETH MØNSTED JOHANSEN, TRANSLATED BY NIELSINE NIELSEN
While Biomarin waits for the US Food and Drug Administration (FDA) to make a decision on the company's gene therapy in hemophilia A, Biomarin publishes five-year highlights of clinical data from studies with the drug.
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