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Long-term evaluation parameters in GNE myopathy: a 5-year observational follow-up natural history study.

Madoka Mori-YoshimuraHiroyuki YajimaYasushi OyaKatsuhiro MizunoSatoru NoguchiIchizo NishinoYuji Takahashi
Published in: BMJ neurology open (2022)
MMT, GMFM, pinch power and %FVC are useful parameters for the long-term evaluation of GNE myopathy patients.
Keyphrases
  • end stage renal disease
  • late onset
  • ejection fraction
  • newly diagnosed
  • chronic kidney disease
  • prognostic factors
  • peritoneal dialysis
  • cross sectional
  • patient reported outcomes
  • early onset
  • muscular dystrophy