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Precision medicine reimagined.

At BioMarin, we have fostered a culture that embraces scientific complexity. We are applying our expertise in molecular medicine to maximize the potential impact of our therapies on how patients feel, function, and thrive.

For instance, we are currently investigating vosoritide, an analog of C-type natriuretic peptide (CNP), for the treatment of children with achondroplasia. Through a comprehensive understanding of the physiological pathways underlying bone development, we discovered that vosoritide is designed to operate in a pathway that is also affected in other conditions associated with impaired bone growth. Vosoritide is now being evaluated in those conditions to determine whether it has potential to serve additional patient communities.

Research into vosoritide is ongoing. It has not been approved for use or determined to be safe or effective.

Image: Normal Bone Growth vs. Bone Growth with Achondroplasia
References

1Horton WA. Growth, Genetics and Hormones. 2006;22:49-54.