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Page 1
Large variation in effects during 10 years of enzyme therapy in adults with Pompe disease.
Harlaar L, Hogrel JY, Perniconi B, Kruijshaar ME, Rizopoulos D, Taouagh N, Canal A, Brusse E, van Doorn PA, van der Ploeg AT, Laforêt P, van der Beek NAME. Harlaar L, et al. Among authors: van der ploeg at, van doorn pa, van der beek name. Neurology. 2019 Nov 5;93(19):e1756-e1767. doi: 10.1212/WNL.0000000000008441. Epub 2019 Oct 16. Neurology. 2019. PMID: 31619483 Free PMC article.
Enzyme replacement therapy in late-onset Pompe's disease: a three-year follow-up.
Winkel LP, Van den Hout JM, Kamphoven JH, Disseldorp JA, Remmerswaal M, Arts WF, Loonen MC, Vulto AG, Van Doorn PA, De Jong G, Hop W, Smit GP, Shapira SK, Boer MA, van Diggelen OP, Reuser AJ, Van der Ploeg AT. Winkel LP, et al. Among authors: van der ploeg at, van diggelen op, van doorn pa, van den hout jm. Ann Neurol. 2004 Apr;55(4):495-502. doi: 10.1002/ana.20019. Ann Neurol. 2004. PMID: 15048888
Long-term intravenous treatment of Pompe disease with recombinant human alpha-glucosidase from milk.
Van den Hout JM, Kamphoven JH, Winkel LP, Arts WF, De Klerk JB, Loonen MC, Vulto AG, Cromme-Dijkhuis A, Weisglas-Kuperus N, Hop W, Van Hirtum H, Van Diggelen OP, Boer M, Kroos MA, Van Doorn PA, Van der Voort E, Sibbles B, Van Corven EJ, Brakenhoff JP, Van Hove J, Smeitink JA, de Jong G, Reuser AJ, Van der Ploeg AT. Van den Hout JM, et al. Among authors: van hirtum h, van hove j, van der ploeg at, van der voort e, van diggelen op, van corven ej, van doorn pa. Pediatrics. 2004 May;113(5):e448-57. doi: 10.1542/peds.113.5.e448. Pediatrics. 2004. PMID: 15121988
Fatigue: an important feature of late-onset Pompe disease.
Hagemans ML, van Schie SP, Janssens AC, van Doorn PA, Reuser AJ, van der Ploeg AT. Hagemans ML, et al. Among authors: van der ploeg at, van schie sp, van doorn pa. J Neurol. 2007 Jul;254(7):941-5. doi: 10.1007/s00415-006-0434-2. Epub 2007 Mar 2. J Neurol. 2007. PMID: 17351726 Free PMC article.
Pompe's disease.
van der Ploeg AT, Reuser AJ. van der Ploeg AT, et al. Lancet. 2008 Oct 11;372(9646):1342-53. doi: 10.1016/S0140-6736(08)61555-X. Lancet. 2008. PMID: 18929906 Review.
PAS-positive lymphocyte vacuoles can be used as diagnostic screening test for Pompe disease.
Hagemans ML, Stigter RL, van Capelle CI, van der Beek NA, Winkel LP, van Vliet L, Hop WC, Reuser AJ, Beishuizen A, van der Ploeg AT. Hagemans ML, et al. Among authors: van der ploeg at, van capelle ci, van der beek na, van vliet l. J Inherit Metab Dis. 2010 Apr;33(2):133-9. doi: 10.1007/s10545-009-9027-4. Epub 2010 Jan 27. J Inherit Metab Dis. 2010. PMID: 20107902 Free PMC article. Clinical Trial.
Lentiviral gene therapy of murine hematopoietic stem cells ameliorates the Pompe disease phenotype.
van Til NP, Stok M, Aerts Kaya FS, de Waard MC, Farahbakhshian E, Visser TP, Kroos MA, Jacobs EH, Willart MA, van der Wegen P, Scholte BJ, Lambrecht BN, Duncker DJ, van der Ploeg AT, Reuser AJ, Verstegen MM, Wagemaker G. van Til NP, et al. Among authors: van der wegen p, van der ploeg at. Blood. 2010 Jul 1;115(26):5329-37. doi: 10.1182/blood-2009-11-252874. Epub 2010 Apr 12. Blood. 2010. PMID: 20385789 Free article.
A randomized study of alglucosidase alfa in late-onset Pompe's disease.
van der Ploeg AT, Clemens PR, Corzo D, Escolar DM, Florence J, Groeneveld GJ, Herson S, Kishnani PS, Laforet P, Lake SL, Lange DJ, Leshner RT, Mayhew JE, Morgan C, Nozaki K, Park DJ, Pestronk A, Rosenbloom B, Skrinar A, van Capelle CI, van der Beek NA, Wasserstein M, Zivkovic SA. van der Ploeg AT, et al. Among authors: van capelle ci, van der beek na. N Engl J Med. 2010 Apr 15;362(15):1396-406. doi: 10.1056/NEJMoa0909859. N Engl J Med. 2010. PMID: 20393176 Free article. Clinical Trial.
250 results