MEK Inhibition in a Newborn with RAF1-Associated Noonan Syndrome Ameliorates Hypertrophic Cardiomyopathy but is Insufficient to Revert Pulmonary Vascular Disease
GENES(2022)
关键词
therapeutics,hypertrophic cardiomyopathy,Noonan syndrome,MEK-inhibitor,RASopathies,transcriptomics
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