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BioMarin Pharmaceutical Inc.

PharmaDeviceNews BioMarin Pharmaceutical Inc.
BioMarin and n-Lorem Foundation are advancing early research into a potential antisense medicine targeting the RNU4-2 variant associated with ReNU syndrome, a newly identified rare neurodevelopmental disorder. Representative image.
Pharma & Biotech

How BioMarin and n-Lorem plan to develop an antisense therapy for ReNU syndrome

BioMarin and n-Lorem launch a preclinical antisense program targeting the RNU4-2 variant behind most ReNU syndrome cases.

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Pharma & Biotech

Could BMN 333 help BioMarin reset expectations in achondroplasia treatment?

Find out how BioMarin’s VOXZOGO and BMN 333 data could reshape rare bone disorder treatment as competition intensifies.

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Representative image of a pediatric growth assessment in a clinical setting. BioMarin’s latest VOXZOGO data highlight how long-term achondroplasia treatment is moving beyond height gains toward arm span, bone health, and broader skeletal development outcomes.
Pharma & Biotech

Why BioMarin’s VOXZOGO update matters ahead of hypochondroplasia Phase 3 data

VOXZOGO is no longer just a height story. BioMarin now needs long-term data to support a broader skeletal dysplasia strategy.

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