Coagulation Laboratory Laurencea Boxer News Today : Breaking News, Live Updates & Top Stories | Vimarsana

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SAN RAFAEL, Calif., Jan. 10, 2021 /PRNewswire/  BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced positive topline results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A.  This is the largest global Phase 3 study to date for any gene therapy in any indication, with 134 participants.  All participants in the study received a single dose of valoctocogene roxaparvovec and completed a year or more of follow-up. 
Figure 1: Box-and-Whiskers Plot of Factor VIII Activity in 4-Week Intervals, Subset Population (N=17 ) - Key: The boxes show the interquartile ranges with the lines in the boxes indicating medians. The ends of the whiskers represent the minimum and maximum values and diamonds indicate the means. - Includes only HIV-negative subjects dosed 2 or more years prior to Nov 2020 data cut date. One subjec ....

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