Long-term evaluation parameters in GNE myopathy: a 5-year observational follow-up natural history study.
Madoka Mori-YoshimuraHiroyuki YajimaYasushi OyaKatsuhiro MizunoSatoru NoguchiIchizo NishinoYuji TakahashiPublished in: BMJ neurology open (2022)
MMT, GMFM, pinch power and %FVC are useful parameters for the long-term evaluation of GNE myopathy patients.