To cite this version:Akinori Uruha, Olivier Benveniste. The prognosis is poor due to severe respiratory insufficiency. Recently, however, autologous stem cell transplantation following high-dose melphalan (HDM-SCT) has been shown to be effective unless there is delay before the treatment. Therefore, early recognition of the disease is important.This review gives an overview of recent advances in SLONM-MGUS, which could help to understand clinical and pathological features and treatment. Autologous stem cell transplantation following high-dose melphalan
Recent findings
Key points• Sporadic late-onset nemaline myopathy (SLONM) with monoclonal gammopathy of undetermined significance (MGUS) shows subacute progressive muscle weakness with severe respiratory insufficiency.• Head drop, MGUS and nemaline body are typical signs of SLONM-MGUS.• Heart failure possibly appears as an extramuscular involvement.• Anti-plasma cell dyscrasia therapy including autologous stem cell transplantation following high-dose melphalan is effective.