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MEK Inhibition in a Newborn with RAF1-Associated Noonan Syndrome Ameliorates Hypertrophic Cardiomyopathy but is Insufficient to Revert Pulmonary Vascular Disease

GENES(2022)

引用 15|浏览28
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关键词
therapeutics,hypertrophic cardiomyopathy,Noonan syndrome,MEK-inhibitor,RASopathies,transcriptomics
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