Effect of Vosoritide Therapy on IGF-I and Endogenous C-Type Natriuretic Peptide in Hypochondroplasia
Authors' conclusion
In children with HCH, IGF-1 levels are reduced and NTproCNP levels are elevated at baseline. A correlation between changes in these levels during treatment suggests a possible interaction between IGF-1 and CNP signaling.
Study context
- Population
- Prepubertal children aged 3 to 11 years with hypochondroplasia and height less than −2.25 SD
- Primary endpoint
- Longitudinal changes in IGF-1 and NTproCNP and their correlations with changes in height SD and AGV
- Follow-up
- 12 months
Methodological detailsunblinded, 6-month baseline observation period
- Unblinded
- 6-month baseline observation period
Primary result
As reported in the abstract
Change in IGF-1 at 12 months positively correlated with change in NTproCNP (ρ)
ρ = 0.57
P = .024
Original publication
Effect of Vosoritide Therapy on IGF-I and Endogenous C-Type Natriuretic Peptide in Hypochondroplasia. J Clin Endocrinol Metab. Published 29 October 2025. doi:10. 1210/ clinem/ dgaf591
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