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Vosoritide Shows Promising Growth Benefits in Children with RASopathies and Related Genetic Short Stature Disorders
Diabetes & Endocrinologygenetic short staturegrowth velocity

Vosoritide Shows Promising Growth Benefits in Children with RASopathies and Related Genetic Short Stature Disorders

By MedXY|Aug 18, 2026

A phase II basket trial demonstrates that vosoritide significantly increases growth velocity in children with RASopathies, ACAN, and NPR2 deficiencies, with an acceptable short-term safety profile, suggesting its potential as a precision th

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