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Year Number of Results
2000 1
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29 results

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Page 1
Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease.
Dimachkie MM, Barohn RJ, Byrne B, Goker-Alpan O, Kishnani PS, Ladha S, Laforêt P, Mengel KE, Peña LDM, Sacconi S, Straub V, Trivedi J, Van Damme P, van der Ploeg AT, Vissing J, Young P, Haack KA, Foster M, Gilbert JM, Miossec P, Vitse O, Zhou T, Schoser B; NEO-EXT investigators. Dimachkie MM, et al. Neurology. 2022 Aug 1;99(5):e536-e548. doi: 10.1212/WNL.0000000000200746. Neurology. 2022. PMID: 35618441 Free PMC article.
Mucopolysaccharidoses and other lysosomal storage diseases.
Lampe C, Bellettato CM, Karabul N, Scarpa M. Lampe C, et al. Among authors: karabul n. Rheum Dis Clin North Am. 2013 May;39(2):431-55. doi: 10.1016/j.rdc.2013.03.004. Rheum Dis Clin North Am. 2013. PMID: 23597973 Review.
Pain in adult patients with Pompe disease.
Karabul N, Kruijshaar ME, Schober A, Güngör D, Hanisch F. Karabul N, et al. Mol Genet Metab Rep. 2014 Apr 2;1:139-140. doi: 10.1016/j.ymgmr.2014.02.007. eCollection 2014. Mol Genet Metab Rep. 2014. PMID: 27896083 Free PMC article. No abstract available.
Motor Development in Infantile Pompe Disease.
Gökce S, Karabul N, Hennermann JB, Mengel E. Gökce S, et al. Among authors: karabul n. J Neuromuscul Dis. 2015;2(s1):S18. J Neuromuscul Dis. 2015. PMID: 27858615 No abstract available.
Treatment of Fabry Disease management with migalastat-outcome from a prospective 24 months observational multicenter study (FAMOUS).
Lenders M, Nordbeck P, Kurschat C, Eveslage M, Karabul N, Kaufeld J, Hennermann JB, Patten M, Cybulla M, Müntze J, Üçeyler N, Liu D, Das AM, Sommer C, Pogoda C, Reiermann S, Duning T, Gaedeke J, von Cossel K, Blaschke D, Brand SM, Mann WA, Kampmann C, Muschol N, Canaan-Kühl S, Brand E. Lenders M, et al. Among authors: karabul n. Eur Heart J Cardiovasc Pharmacother. 2022 May 5;8(3):272-281. doi: 10.1093/ehjcvp/pvab025. Eur Heart J Cardiovasc Pharmacother. 2022. PMID: 35512362
Pregnancy and delivery in women with Pompe disease.
Karabul N, Berndt J, Kornblum C, Kley RA, Wenninger S, Tiling N, Mengel E, Plöckinger U, Vorgerd M, Deschauer M, Schoser B, Hanisch F. Karabul N, et al. Mol Genet Metab. 2014 Jun;112(2):148-53. doi: 10.1016/j.ymgme.2014.03.010. Epub 2014 Mar 30. Mol Genet Metab. 2014. PMID: 24726296
Outcome of patients with classical infantile pompe disease receiving enzyme replacement therapy in Germany.
Hahn A, Praetorius S, Karabul N, Dießel J, Schmidt D, Motz R, Haase C, Baethmann M, Hennermann JB, Smitka M, Santer R, Muschol N, Meyer A, Marquardt T, Huemer M, Thiels C, Rohrbach M, Seyfullah G, Mengel E. Hahn A, et al. Among authors: karabul n. JIMD Rep. 2015;20:65-75. doi: 10.1007/8904_2014_392. Epub 2015 Jan 28. JIMD Rep. 2015. PMID: 25626711 Free PMC article.
Treatment of Fabry's Disease With Migalastat: Outcome From a Prospective Observational Multicenter Study (FAMOUS).
Lenders M, Nordbeck P, Kurschat C, Karabul N, Kaufeld J, Hennermann JB, Patten M, Cybulla M, Müntze J, Üçeyler N, Liu D, Das AM, Sommer C, Pogoda C, Reiermann S, Duning T, Gaedeke J, Stumpfe K, Blaschke D, Brand SM, Mann WA, Kampmann C, Muschol N, Canaan-Kühl S, Brand E. Lenders M, et al. Among authors: karabul n. Clin Pharmacol Ther. 2020 Aug;108(2):326-337. doi: 10.1002/cpt.1832. Epub 2020 Apr 27. Clin Pharmacol Ther. 2020. PMID: 32198894
29 results