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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: rizopoulos d. Neurology. 2019 Nov 5;93(19):e1756-e1767. doi: 10.1212/WNL.0000000000008441. Epub 2019 Oct 16. Neurology. 2019. PMID: 31619483 Free PMC article.
Quality of life and participation in daily life of adults with Pompe disease receiving enzyme replacement therapy: 10 years of international follow-up.
Güngör D, Kruijshaar ME, Plug I, Rizopoulos D, Kanters TA, Wens SC, Reuser AJ, van Doorn PA, van der Ploeg AT. Güngör D, et al. Among authors: rizopoulos d. J Inherit Metab Dis. 2016 Mar;39(2):253-60. doi: 10.1007/s10545-015-9889-6. Epub 2015 Nov 3. J Inherit Metab Dis. 2016. PMID: 26531313 Free PMC article.
Pompe disease in adulthood: effects of antibody formation on enzyme replacement therapy.
de Vries JM, Kuperus E, Hoogeveen-Westerveld M, Kroos MA, Wens SC, Stok M, van der Beek NA, Kruijshaar ME, Rizopoulos D, van Doorn PA, van der Ploeg AT, Pijnappel WW. de Vries JM, et al. Among authors: rizopoulos d. Genet Med. 2017 Jan;19(1):90-97. doi: 10.1038/gim.2016.70. Epub 2016 Jun 30. Genet Med. 2017. PMID: 27362911 Free article.
Response to Herbert et al.
de Vries JM, Kuperus E, Hoogeveen-Westerveld M, Kroos MA, Wens SCA, Stok M, van der Beek NAME, Kruijshaar ME, Rizopoulos D, van Doorn PA, van der Ploeg AT, Pijnappel WWMP. de Vries JM, et al. Among authors: rizopoulos d. Genet Med. 2017 Nov;19(11):1283-1284. doi: 10.1038/gim.2017.49. Epub 2017 Jun 29. Genet Med. 2017. PMID: 28661488 Free article. No abstract available.
214 results