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STIG study: real-world data of long-term outcomes of adults with Pompe disease under enzyme replacement therapy with alglucosidase alfa.
Gutschmidt K, Musumeci O, Díaz-Manera J, Chien YH, Knop KC, Wenninger S, Montagnese F, Pugliese A, Tavilla G, Alonso-Pérez J, Hwu PW, Toscano A, Schoser B. Gutschmidt K, et al. Among authors: hwu pw. J Neurol. 2021 Jul;268(7):2482-2492. doi: 10.1007/s00415-021-10409-9. Epub 2021 Feb 5. J Neurol. 2021. PMID: 33543425 Free PMC article.
Corrigendum to "Evaluation of 3-O-methyldopa as a biomarker for aromatic L-amino acid decarboxylase deficiency in 7 Brazilian cases" [27/100744/2021/ pages: 1-4].
Kubaski F, Herbst ZM, Pereira DAA, Silva C, Chen C, Hwu PWL, van der Linden H, Lourenço CM, Giugliani R. Kubaski F, et al. Among authors: hwu pwl. Mol Genet Metab Rep. 2022 Nov 23;33:100943. doi: 10.1016/j.ymgmr.2022.100943. eCollection 2022 Dec. Mol Genet Metab Rep. 2022. PMID: 38234304 Free PMC article.