What question did this study set out to answer?

This research aims to evaluate the effectiveness of vosoritide in enhancing growth in children with genetic conditions related to the Ras-MAPK pathway.

April 14, 2026The Journal of Clinical Endocrinology & MetabolismOpen Access

A Phase II Basket Trial of Vosoritide in Children with RASopathies, ACAN and NPR2 Deficiency

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This research aims to evaluate the effectiveness of vosoritide in enhancing growth in children with genetic conditions related to the Ras-MAPK pathway.
Conducted a prospective phase 2 trial in an academic medical center.
Included thirty pre-pubertal children aged 3 to 11 with RASopathies, ACAN, or NPR2 deficiency.
Implemented a 6-month observation period followed by 12-month treatment with vosoritide (15 micrograms/kg/day).
Measured outcomes included annualized growth velocity and height standard deviation scores.
Growth velocity increased significantly from 4.53 cm/yr to 8.09 cm/yr after treatment (p<0.0001).
Age and sex-adjusted AGV Z-score improved by 4.0 SD (95% CI 3.08-4.91) (p<0.0001).
Height increased by 0.65 SD (95% CI 0.53-0.77) during the treatment period (p<0.0001).
Short-term safety profile was good, with mild injection reactions reported as the most common adverse events.

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Implication

Prospective trial shows vosoritide improves growth in children with RASopathies and related deficiencies, suggesting treatment efficacy.

Cite This Study

Dauber et al. (2026) studied this question.

synapsesocial.com/papers/69ddda22e195c95cdefd7a66 https://doi.org/10.1210/clinem/dgag157

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A Phase II Basket Trial of Vosoritide in Children with RASopathies, ACAN and NPR2 Deficiency

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Discussion

Implication

Cite This Study

A Phase II Basket Trial of Vosoritide in Children with RASopathies, ACAN and NPR2 Deficiency

Key Points

Discussion

Implication

Cite This Study

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