Effects of short-to-long term enzyme replacement therapy (ERT) on skeletal muscle tissue in late onset Pompe disease (LOPD).

Michela RipoloneR ViolanoD RonchiS MondelloA NascimbeniI ColomboG FagiolariA BordoniF FortunatoV LucchiniS SarediM FilostoO MusumeciP ToninT MonginiS PrevitaliL MorandiC AngeliniM MoraM SandriM SciaccoA ToscanoG P ComiM Moggio

Published in: Neuropathology and applied neurobiology (2017)

ERT positively modified skeletal muscle pathology as well as motor and respiratory outcomes in the majority of LOPD patients.

Keyphrases

late onset
skeletal muscle
replacement therapy
early onset
end stage renal disease
insulin resistance
smoking cessation
chronic kidney disease
newly diagnosed
ejection fraction
peritoneal dialysis
prognostic factors
type diabetes
adipose tissue
metabolic syndrome
patient reported outcomes
respiratory tract