At the just concluded AOMC meeting in Xi’an, Prof. NISHINO from Japan proposed the idea of performing bone marrow transplantation for the treatment of GNE myopathy, which is very rare. This disease was formerly called distal myopathy accompanied by encrusted vacuoles and was renamed GNE myopathy in 2012 because this disease also manifests with proximal muscle weakness. Since it is difficult for pharmaceutical companies to fund research and production of drugs for this disease, a more feasible approach is bone marrow transplantation, which solves the problem once and for all, and bone marrow cells have been found to secrete the GNE enzyme. Bone marrow transplantation is different from stem cell therapy, which currently in China generally survives no more than 1 week and does not solve the problem. The high cost and risk of bone marrow transplantation is also estimated to be impractical, and an easier way to treat this disease is needed.