Clinical insights in enzyme replacement therapy for metabolic storage disorders: lessons from Pompe disease
Nadine A. M. E. van der Beek, Maudy T M Theunissen, Johanna M. P. van den Hout, W.W.M. Pim Pijnappel, Benedikt Schoser, Pascal Laforêt, Giancarlo Parenti, Pieter A. van Doorn, Ans T. van der Ploeg
Topics & Concepts
Enzyme replacement therapyDiseaseMedicineIntensive care medicineInternal medicineLysosomal Storage Disorders ResearchGlycogen Storage Diseases and MyoclonusChild Nutrition and Feeding Issues