BioMarin Pharmaceutical Inc. announced new data from studies of Voxzogo (vosoritide), demonstrating meaningful impact on tibial bowing in children with achondroplasia and investigational early efficacy results in other skeletal… read more.
BioMarin Pharmaceutical Inc. announced that the Phase III PEGASUS trial evaluating Palynziq (pegvaliase-pqpz) met its primary efficacy endpoint, demonstrating a statistically significant lowering in blood Phe levels in adolescents… read more.
BioMarin Pharmaceutical Inc. announced that the FDA has approved the supplemental New Drug Application (sNDA) for Voxzogo (vosoritide) to increase linear growth in pediatric patients with achondroplasia with… read more.
BioMarin Pharmaceutical Inc. 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… read more.
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