36-Months follow-up assessment after cessation and resuming of enzyme replacement therapy in late onset Pompe disease: data from the Swiss Pompe Registry.

Olivier ScheideggerDaniela LeupoldRafael SauterOliver FindlingKai Michael RöslerThomas Hundsberger

Published in: Journal of neurology (2018)

Our data suggests that long-term interruption of ERT in LOPD may lead to deterioration of clinical meaningful parameters and quality of life. In addition, a clinical restoration after ERT cessation is possible for most of the LOPD patients within a 36 months follow-up.

Keyphrases

late onset
replacement therapy
early onset
end stage renal disease
smoking cessation
electronic health record
ejection fraction
newly diagnosed
chronic kidney disease
peritoneal dialysis
big data
prognostic factors
data analysis
clinical evaluation