A Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment

Study Summary

The purpose of this basket study in children with Turner syndrome, SHOX deficiency, and Noonan syndrome is to evaluate the effect of 3 doses of vosoritide on growth as measured by AGV after 6 months of treatment. The long-term efficacy and safety of vosoritide at the therapeutic dose will be evaluated up to FAH.

Conditions

Short Stature Homeobox- Containing Gene SHOX Deficiency Noonan Syndrome Turner Syndrome

Interventions / Drugs

Vosoritide Injection

View on ClinicalTrials.gov ↗

Sponsor

BMRN

BioMarin Pharmaceutical

Rare Disease

D36/100

Study Details

Enrollment54 participants

Primary Completion—

Lead SponsorBioMarin Pharmaceutical

Data Retrieved2026-04-11 02:55:23.531568+00:00

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