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Six-year study finds bone health maintained in children on VOXZOGO
BioMarin said annual DXA scans in 119 children showed bone mineral content rose over six years while bone density scores stayed steady.
- On Saturday, BioMarin Pharmaceutical Inc. presented research at the Pediatric Endocrine Society's 2026 Annual Meeting in San Francisco, demonstrating VOXZOGO's positive long-term impact on arm span and bone density in children with achondroplasia.
- Achondroplasia, which affects approximately 1 in 25,000 live births, results from a genetic mutation that negatively regulates bone growth; VOXZOGO acts as a positive regulator to promote endochondral bone development.
- A study of 119 children showed that bone mineral content increased over time while bone mineral density Z-scores remained consistent, indicating treatment maintained proportional skeletal health.
- Topline results from the Phase 3 clinical trial for children with hypochondroplasia are expected in the first half of 2026, with BioMarin planning to submit data to health authorities later that year.
- Greg Friberg, Executive Vice President and Chief Research & Development Officer at BioMarin, stated the company remains committed to understanding long-term benefits, with over ten years of clinical research now supporting the drug's use.
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Helena Independent Record+18 Reposted by 18 other sources
BioMarin Presents New Data on the Effect of Long-Term Treatment with VOXZOGO® (vosoritide) on Arm Span, Bone Health and Growth in Children With Achondroplasia at the Pediatric Endocrine Society's 2026 Annual Meeting
VOXZOGO is the only approved treatment for children with achondroplasia starting at birth, with over 10 years of clinical research demonstrating the long-term benefit on complications associated with achondroplasia
·Helena, United States
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Leaning Left2Leaning Right0Center11Last UpdatedBias Distribution85% Center
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