Case report highlights co-occurrence of sporadic late-onset nemaline myopathy and glycogen accumulation responsive to immunotherapy, suggesting new treatment options.
This case expands the MGAM spectrum, highlighting co-occurrence of SLONM and an excessive glycogen accumulation responsive to immunotherapy and transplantation. These findings suggest a spectrum of related disease processes. Recognition of this combined pathology is clinically important, as affected patients may benefit from targeted immunotherapy or stem cell transplantation.
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Jones et al. (2026) studied this question.
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