New Study Results Highlight BioMarin's VOXZOGO Efficacy in Children

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is among the best stocks with the highest upside potential. According to the announcement on September 8, 2025, BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) presented new data from 14 studies at the American Society for Bone and Mineral Research 2025 (ASBMR) Annual Meeting in Seattle. The presentation also outlined the results of treatment with VOXZOGO (vosoritide), highlighting both anatomical improvements in spinal morphology and continued efficacy in children who received the medicine.

An evaluation of the double-blind and placebo-controlled Phase 2 CANOPY clinical study (111-206), conducted in children aged 5 or under with achondroplasia, analyzed the effect of VOXZOGO on spinal morphology, a factor that is a root of spinal stenosis, a serious complication of the condition, where clinical data is still inadequate.

If there’s one company that everyone has high hopes for, it’s BioMarin Pharmaceutical Inc. (NASDAQ:BMRN). Although the company delivered negative returns in the past, the future doesn’t quite appear the same, all thanks to the Inozyme acquisition, which is anticipated to secure what is often called “a first mover advantage.”

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is a California-based biotechnology company that develops and commercializes therapies for life-threatening diseases. Incorporated in 1996, the company is dedicated to pursuing bold scientific endeavors.

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