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“The CMR substudy is the first randomized, controlled clinical trial of HCM patients to show that a once-daily oral therapeutic agent can reduce left ventricular wall thickness and mass, and positively impact several other parameters of cardiac structure and function. Left ventricular hypertrophy and left atrial volumes in particular are predictive of poor prognosis in HCM patients, so it will be exciting to see how mavacamten impacts cardiac function and disease progression as we continue to follow patients over time.”
“Improvement in the key echocardiographic features of hypertrophic cardiomyopathy supports the hypothesis that mavacamten can be used as a disease-specific therapy, which would be a significant advance in therapy for this population. These findings reinforced and extended data from prior open label trials. Additional changes in measures of cardiac structure and function were also observed including reduction in the size of the left atrium. Together, these results reflect this medication's impact on the underlying pathophysiology of hypertrophic cardiomyopathy.”
“Data from the CMR substudy complements and extends our observations of mavacamten's positive benefits on cardiac function and symptom relief, providing us with encouraging evidence that mavacamten treatment may change the course of the adverse cardiac remodeling inherent to HCM. In directly targeting the proteins of the heart muscle that lead to the excessive contraction and impaired relaxation underlying HCM, we have a growing body of data showing that mavacamten is reducing hypercontractility and hypertrophy and reducing harm to the cardiac muscle while maintaining contractile function within the normal range.”
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Saberi et al. (2020) studied this question.
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