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- W2755513263 abstract "Sporadic late onset nemaline myopathy (SLONM) is a rare subacute progressive myopathy which muscle pathology play the crucial role in diagnosis. Without appropriate treatment, the prognosis of SLONM associated with monoclonal gammopathy of undetermined significance (MGUS) is unfavorable. Effective treatment regimens reported in the literature include high-dose intravenous mephalan with autologous stem cell transplantation and immunotherapy. We report a case of 33-year-old man with a 5-month history of proximal muscle weakness who later develop neck weakness and require tracheostomy. SLONM is diagnosed on muscle biopsy. MGUS of IgG lambda type is later identified. The patient is treated with a regimen including cyclophosphamide, thalidomide, and dexamethasone (CTD) and show dramatic clinical response after the 5th cycle. CTD can be considered as alternative regimen for SLONM associated with MGUS in a limited resource situation." @default.
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- W2755513263 date "2017-10-01" @default.
- W2755513263 modified "2023-09-25" @default.
- W2755513263 title "Cyclophosphamide, thalidomide, and dexamethasone as alternative treatment regimen for sporadic late onset nemaline myopathy associated with monoclonal gammopathy of undetermined significance" @default.
- W2755513263 doi "https://doi.org/10.1016/j.nmd.2017.06.241" @default.
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