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11 p, 1.0 MB STIG study : real-world data of long-term outcomes of adults with Pompe disease under enzyme replacement therapy with alglucosidase alfa / Gutschmidt, K. (Department of Neurology. Friedrich-Baur-Institute. Ludwig-Maximilians University Munich) ; Musumeci, O. (Department of Clinical and Experimental Medicine. University of Messina) ; Diaz-Manera, Jordi (Institut d'Investigació Biomèdica Sant Pau) ; Chien, Y.H. (Department of Medical Genetics and Pediatrics. National Taiwan University Hospital) ; Knop, K.C. (Neurologische Praxis Neuer Wall) ; Wenninger, S. (Department of Neurology. Friedrich-Baur-Institute. Ludwig-Maximilians University Munich) ; Montagnese, F. (Department of Neurology. Friedrich-Baur-Institute. Ludwig-Maximilians University Munich) ; Pugliese, A. (Department of Clinical and Experimental Medicine. University of Messina) ; Tavilla, G. (Department of Clinical and Experimental Medicine. University of Messina) ; Alonso-Pérez, Jorge (Institut d'Investigació Biomèdica Sant Pau) ; Hwu, P.W.L. (Department of Medical Genetics and Pediatrics. National Taiwan University Hospital) ; Toscano, A. (Department of Clinical and Experimental Medicine. University of Messina) ; Schoser, B. (Department of Neurology. Friedrich-Baur-Institute. Ludwig-Maximilians University Munich)
Background: Pompe disease is one of the few neuromuscular diseases with an approved drug therapy, which has been available since 2006. Our study aimed to determine the real-world long-term efficacy and safety of alglucosidase alfa. [...]
2021 - 10.1007/s00415-021-10409-9
Journal of Neurology, Vol. 268 Núm. 7 (july 2021) , p. 2482-2492  
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