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BioMarin Announces Positive Phase 3 Gene Therapy Trial Results in Adults with Severe Hemophilia A; Study Met All Primary and Secondary Efficacy Endpoints in One-Year Data Set

Share this article Share this article SAN RAFAEL, Calif., Jan. 10, 2021 /PRNewswire/ --ย 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.ย  This is the largest global Phase 3 study to date for any gene therapy in any indication, with 134 participants. ย All participants in the study received a sing...
United States University Of Southern California Los Angeles Hank Fuchs Md Research Professor Of Pediatrics Biomarin Pharmaceutical Inc

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