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

PR Newswire 18 May 2026
ENERGY 3 study met 1 of 2 co-primary endpoints in children with ENPP1 deficiency ... Deficiency in ENPP1 function results in a decrease in plasma inorganic pyrophosphate (PPi) that can lead to progressive damage to blood vessels, soft tissues and bones.
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