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Achondroplasia is an autosomal dominant condition with growth failure due to inhibition of endochondral ossification. In an industry-funded, phase II study, researchers evaluated the safety and dose of a once-daily subcutaneous injection of vosoritide, a C-type natriuretic peptide analogue known to stimulate endochondral ossification, in 35 patients aged 5 to 14 years. Participants were divided into four cohorts by dose (2.5, 7.5, 15, and 30 µg/kg of body weight). In the 2.5- and 7.5-µg/kg cohorts, doses were escalated after 6 months to a maximum dose of 15 µg/kg. Outcomes were assessed up to 42 months.
Growth velocity increased for all cohorts throughout the study, and in a dose-dependent pattern during the first 6 months for those receivin…