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Modelling the neuropathology of lysosomal storage disorders through disease-specific human induced pluripotent stem cells.
Kobolák J, Molnár K, Varga E, Bock I, Jezsó B, Téglási A, Zhou S, Lo Giudice M, Hoogeveen-Westerveld M, Pijnappel WP, Phanthong P, Varga N, Kitiyanant N, Freude K, Nakanishi H, László L, Hyttel P, Dinnyés A. Kobolák J, et al. Among authors: pijnappel wp. Exp Cell Res. 2019 Jul 15;380(2):216-233. doi: 10.1016/j.yexcr.2019.04.021. Epub 2019 Apr 27. Exp Cell Res. 2019. PMID: 31039347 Free article.
European consensus for starting and stopping enzyme replacement therapy in adult patients with Pompe disease: a 10-year experience.
van der Ploeg AT, Kruijshaar ME, Toscano A, Laforêt P, Angelini C, Lachmann RH, Pascual Pascual SI, Roberts M, Rösler K, Stulnig T, van Doorn PA, Van den Bergh PYK, Vissing J, Schoser B; European Pompe Consortium. van der Ploeg AT, et al. Eur J Neurol. 2017 Jun;24(6):768-e31. doi: 10.1111/ene.13285. Epub 2017 May 6. Eur J Neurol. 2017. PMID: 28477382 Review.