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Lentiviral Stem Cell Gene Therapy for Pompe Disease.
Liang Q, Stok M, van Helsdingen Y, van der Velden G, Jacobs E, Duncker D, Reuser A, van der Ploeg A, Vulto A, van Til NP, Wagemaker G. Liang Q, et al. J Neuromuscul Dis. 2015;2(s1):S64. J Neuromuscul Dis. 2015. PMID: 27858653 No abstract available.
The ACE I/D polymorphism does not explain heterogeneity of natural course and response to enzyme replacement therapy in Pompe disease.
Kuperus E, van der Meijden JC, In 't Groen SLM, Kroos MA, Hoogeveen-Westerveld M, Rizopoulos D, Martinez MYN, Kruijshaar ME, van Doorn PA, van der Beek NAME, van der Ploeg AT, Pijnappel WWMP. Kuperus E, et al. Among authors: van der ploeg at, van der meijden jc, van doorn pa, van der beek name. PLoS One. 2018 Dec 7;13(12):e0208854. doi: 10.1371/journal.pone.0208854. eCollection 2018. PLoS One. 2018. PMID: 30532252 Free PMC article. Clinical Trial.
Discontinuation of enzyme replacement therapy in adults with Pompe disease: Evaluating the European POmpe Consortium stop criteria.
van Kooten HA, Harlaar L, van der Beek NAME, van Doorn PA, van der Ploeg AT, Brusse E; Erasmus MC Pompe expert committee. van Kooten HA, et al. Among authors: van der ploeg at, van doorn pa, van der beek name. Neuromuscul Disord. 2020 Jan;30(1):59-66. doi: 10.1016/j.nmd.2019.11.007. Epub 2019 Nov 22. Neuromuscul Disord. 2020. PMID: 31911071 Free article.
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 ploeg at, van der giessen lj, van den hout jmp, van den dorpel jja, van der beek name. 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.
IGF2-tagging of GAA promotes full correction of murine Pompe disease at a clinically relevant dosage of lentiviral gene therapy.
Liang Q, Catalano F, Vlaar EC, Pijnenburg JM, Stok M, van Helsdingen Y, Vulto AG, van der Ploeg AT, van Til NP, Pijnappel WWMP. Liang Q, et al. Among authors: van helsdingen y, van der ploeg at, van til np. Mol Ther Methods Clin Dev. 2022 Sep 24;27:109-130. doi: 10.1016/j.omtm.2022.09.010. eCollection 2022 Dec 8. Mol Ther Methods Clin Dev. 2022. PMID: 36284764 Free PMC article.
Tagged IDS causes efficient and engraftment-independent prevention of brain pathology during lentiviral gene therapy for Mucopolysaccharidosis type II.
Catalano F, Vlaar EC, Katsavelis D, Dammou Z, Huizer TF, van den Bosch JC, Hoogeveen-Westerveld M, van den Hout HJMP, Oussoren E, Ruijter GJG, Schaaf G, Pike-Overzet K, Staal FJT, van der Ploeg AT, Pijnappel WWMP. Catalano F, et al. Among authors: van der ploeg at. Mol Ther Methods Clin Dev. 2023 Nov 2;31:101149. doi: 10.1016/j.omtm.2023.101149. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38033460 Free PMC article.
Seven cases of Pompe disease from Greece.
Kroos M, Manta P, Mavridou I, Muntoni F, Halley D, Van der Helm R, Zaifeiriou D, Van der Ploeg A, Reuser A, Michelakakis H. Kroos M, et al. J Inherit Metab Dis. 2006 Aug;29(4):556-63. doi: 10.1007/s10545-006-0280-5. J Inherit Metab Dis. 2006. PMID: 16838077
243 results