BioMarin Provides Update on Phase 3 Trial for BMN 401 in Children Aged 1-12 With ENPP1 Deficiency

ENERGY 3 study met 1 of 2 co-primary endpoints in children with ENPP1 deficiency Treatment with BMN 401 led to statistically significant increases in plasma inorganic pyrophosphate (PPi) concentration, one of the study's co-primary endpoints; however, no improvement was observed in...