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CO

Christine O'Brien

GACI Global Co-President

BioMarin Pharmaceutical

BioMarin Pharmaceutical Pipeline

DrugIndicationPhase
VOXZOGO (vosoritide)AchondroplasiaCommercial
PALYNZIQ (pegvaliase-pqpz)Phenylketonuria (PKU)Commercial
Enzyme Replacement TherapyMucopolysaccharidosis ICommercial
Gene TherapyHemophilia ACommercial
Metabolic TherapyRare Metabolic ConditionCommercial
ENPP1 deficiency programENPP1 deficiency/GACIClinical