Trial reportThe Journal of clinical endocrinology and metabolism2026
A phase II basket trial of vosoritide in children with RASopathies, ACAN, and NPR2 deficiency.
Trial report in The Journal of clinical endocrinology and metabolism, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
2 citing papers in PubMed.
- A phase II basket trial of vosoritide in children with RASopathies, ACAN, and NPR2 deficiency.The Journal of clinical endocrinology and metabolism · 2026Trial
- Emerging NPR-B agonists for cartilage and bone: from CNP biology to translational compound design.Frontiers in chemistry · 2026Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
8 authors.
Funding
Abstract
contextGenetic defects in many biological pathways, including activation of the Ras-MAPK pathway, cause short stature. Vosoritide, a C-type natriuretic peptide analog that inhibits this pathway, has been approved for use in achondroplasia.
objectiveTo determine whether vosoritide improves growth in children with disorders of the Ras-MAPK pathway, including RASopathies, ACAN, and NPR2 deficiency.
designProspective Phase 2 basket trial.
settingAcademic medical center.
participantsThirty prepubertal children aged 3 to 11 years with a RASopathy, ACAN or NPR2 deficiency and height ≤ -2.25 SD.
interventionSix-month observation period followed by 12-month treatment with vosoritide subcutaneously 15 µg/kg/day.
main outcome measuresCo-primary outcomes included incidence of adverse events, change in annualized growth velocity (AGV), and height standard deviation scores.
resultsThe AGV increased from 4.53 ± 1.61 cm/year to 8.09 ± 1.58 cm/year with treatment (P < .0001). This corresponded to a 4.0 SD (95%CI 3.08-4.91) increase in age and sex-adjusted AGV Z-score (P < .0001). The increase in AGV was seen in all genetic subgroups. There was a height increase of 0.65 SD (95%CI 0.53-0.77) in the treatment vs observation period (P < .0001).Short-term safety was reassuring, with mild injection site reactions being the most common adverse events. However, with longer use, 5 subjects discontinued medication due to adverse events, including 3 slipped capital femoral epiphyses and 4 cases of genu valgum.
conclusionVosoritide led to marked increases in growth velocity in children with RASopathies, ACAN, and NPR2 deficiency, raising the possibility that vosoritide could be an effective precision medicine for all growth disorders affecting the MAPK pathway.
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