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Year Number of Results
2001 1
2007 2
2013 1
2017 1
2020 2
2021 0
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Page 1
Effects of higher and more frequent dosing of alglucosidase alfa and immunomodulation on long-term clinical outcome of classic infantile Pompe patients.
Poelman E, van den Dorpel JJA, Hoogeveen-Westerveld M, van den Hout JMP, van der Giessen LJ, van der Beek NAME, Pijnappel WWMP, van der Ploeg AT. Poelman E, et al. Among authors: van der giessen lj. J Inherit Metab Dis. 2020 Nov;43(6):1243-1253. doi: 10.1002/jimd.12268. Epub 2020 Jul 13. J Inherit Metab Dis. 2020. PMID: 32506446 Free PMC article.
Distal muscle weakness is a common and early feature in long-term enzyme-treated classic infantile Pompe patients.
van den Dorpel JJA, Poelman E, Harlaar L, van Kooten HA, van der Giessen LJ, van Doorn PA, van der Ploeg AT, van den Hout JMP, van der Beek NAME. van den Dorpel JJA, et al. Among authors: van der giessen lj. Orphanet J Rare Dis. 2020 Sep 14;15(1):247. doi: 10.1186/s13023-020-01482-w. Orphanet J Rare Dis. 2020. PMID: 32928284 Free PMC article.