STIG study: real-world data of long-term outcomes of adults with Pompe disease under enzyme replacement therapy with alglucosidase alfa

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. METHODS: This multicenter retrospective study (NCT02824068) collecte...

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Bibliografske podrobnosti
izdano v:J Neurol
Main Authors: Gutschmidt, Kristina, Musumeci, Olimpia, Díaz-Manera, Jordi, Chien, Yin-Hsiu, Knop, Karl Christian, Wenninger, Stephan, Montagnese, Federica, Pugliese, Alessia, Tavilla, Graziana, Alonso-Pérez, Jorge, Hwu, Paul Wuh-Liang, Toscano, Antonio, Schoser, Benedikt
Format: Artigo
Jezik:Inglês
Izdano: Springer Berlin Heidelberg 2021
Teme:
Original Communication
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC7862044/
https://ncbi.nlm.nih.gov/pubmed/33543425
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s00415-021-10409-9
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