Key result
A 3-year-old boy with MYPN-mutated nemaline myopathy developed severe dilated cardiomyopathy and refractory heart failure, ultimately dying 4 months after admission despite medical therapy.
Case Report (n=1)
Nemaline myopathy can present with severe dilated cardiomyopathy and refractory heart failure, for which heart transplantation may be considered as a potential solution.
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May support transplant evaluation in refractory genetic NM cardiomyopathy; hypothesis-generating and should not yet change practice.
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Wang et al. (2021) conducted a case report in Nemaline myopathy with dilated cardiomyopathy and severe heart failure (n=1). Medical management for heart failure was evaluated on Clinical outcome. A 3-year-old boy with MYPN-mutated nemaline myopathy developed severe dilated cardiomyopathy and refractory heart failure, ultimately dying 4 months after admission despite medical therapy.
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