Rigosertib Reverses Hypertrophic Cardiomyopathy in Noonan Syndrome.

Rigosertib reverses hypertrophic cardiomyopathy and other Noonan syndrome features in animal models, showing promise as a targeted therapy for RASopathies.

• Why it matters: RASopathies like Noonan syndrome cause serious developmental and cardiac issues with limited treatment options, necessitating new therapeutic strategies to improve patient outcomes.
• What they did: Researchers screened drugs in Drosophila models expressing RASopathy genes and validated findings in Raf1 L613V/+ mice, assessing cardiac and syndromic features through molecular and imaging techniques.
• The result: Treatment with rigosertib significantly reversed cardiac hypertrophy, normalized gene expression, and improved skeletal and craniofacial abnormalities, indicating its potential to treat RASopathy-related pathologies.