Does once-daily subcutaneous vosoritide improve annualized growth velocity in children with achondroplasia?
Once-daily subcutaneous vosoritide provides a sustained increase in annualized growth velocity with a mild side-effect profile in children with achondroplasia.
In children with achondroplasia, once-daily subcutaneous administration of vosoritide was associated with a side-effect profile that appeared generally mild. Treatment resulted in a sustained increase in the annualized growth velocity for up to 42 months. (Funded by BioMarin Pharmaceutical; ClinicalTrials.gov numbers, NCT01603095, NCT02055157, and NCT02724228.).
Savarirayan et al. (Tue,) studied this question.