RASopathies

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CardiologyHypertrophic CardiomyopathyRASopathies

Rigosertib: A Promising Therapeutic Agent for Reversing Hypertrophic Cardiomyopathy in Noonan Syndrome

By MedXY|Aug 19, 2026

Rigosertib demonstrates significant reversal of hypertrophic cardiomyopathy and other syndromic features in Noonan syndrome models, highlighting its potential as a targeted therapy for RASopathy-associated cardiac and developmental abnormal

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