SAN RAFAEL, Calif., March 8, 2021 /PRNewswire/-- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene roxaparvovec, an investigational gene therapy for th...
BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that an individual in Germany with severe hemophilia A was treated with ROCTAVIAN® (valoctocogene roxaparvovec-rvox), marking the first time that the gene therapy has been g...
Patients less than 37 weeks post-menstrual age (gestational at birth plus post-natal age) or those weighing less than 2.5kg:BRINEURA is not recommended for use in these patients due to physiologic immaturity which may increase risk of serious and clinically significant adverse reactions observe...