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A Phase 3, multicenter, open-label, switchover trial to assess the safety and efficacy of taliglucerase alfa, a plant cell-expressed recombinant human glucocerebrosidase, in adult and pediatric patients with Gaucher disease previously treated with imiglucerase.
Pastores GM, Petakov M, Giraldo P, Rosenbaum H, Szer J, Deegan PB, Amato DJ, Mengel E, Tan ES, Chertkoff R, Brill-Almon E, Zimran A. Pastores GM, et al. Among authors: szer j. Blood Cells Mol Dis. 2014 Dec;53(4):253-60. doi: 10.1016/j.bcmd.2014.05.004. Epub 2014 Jun 18. Blood Cells Mol Dis. 2014. PMID: 24950666 Clinical Trial.
Enzyme replacement therapy with taliglucerase alfa: 36-month safety and efficacy results in adult patients with Gaucher disease previously treated with imiglucerase.
Pastores GM, Shankar SP, Petakov M, Giraldo P, Rosenbaum H, Amato DJ, Szer J, Chertkoff R, Brill-Almon E, Zimran A. Pastores GM, et al. Among authors: szer j. Am J Hematol. 2016 Jul;91(7):661-5. doi: 10.1002/ajh.24399. Epub 2016 May 18. Am J Hematol. 2016. PMID: 27102949 Free PMC article. Clinical Trial.
Classifying the additional morbidities of Gaucher disease.
Langeveld M, Elstein D, Szer J, Hollak CEM, Zimran A. Langeveld M, et al. Among authors: szer j. Blood Cells Mol Dis. 2018 Feb;68:209-210. doi: 10.1016/j.bcmd.2016.12.006. Epub 2016 Dec 19. Blood Cells Mol Dis. 2018. PMID: 28024892 No abstract available.
Management goals for type 1 Gaucher disease: An expert consensus document from the European working group on Gaucher disease.
Biegstraaten M, Cox TM, Belmatoug N, Berger MG, Collin-Histed T, Vom Dahl S, Di Rocco M, Fraga C, Giona F, Giraldo P, Hasanhodzic M, Hughes DA, Iversen PO, Kiewiet AI, Lukina E, Machaczka M, Marinakis T, Mengel E, Pastores GM, Plöckinger U, Rosenbaum H, Serratrice C, Symeonidis A, Szer J, Timmerman J, Tylki-Szymańska A, Weisz Hubshman M, Zafeiriou DI, Zimran A, Hollak CEM. Biegstraaten M, et al. Among authors: szer j. Blood Cells Mol Dis. 2018 Feb;68:203-208. doi: 10.1016/j.bcmd.2016.10.008. Epub 2016 Oct 24. Blood Cells Mol Dis. 2018. PMID: 28274788 Free article.
Exploring the patient journey to diagnosis of Gaucher disease from the perspective of 212 patients with Gaucher disease and 16 Gaucher expert physicians.
Mehta A, Belmatoug N, Bembi B, Deegan P, Elstein D, Göker-Alpan Ö, Lukina E, Mengel E, Nakamura K, Pastores GM, Pérez-López J, Schwartz I, Serratrice C, Szer J, Zimran A, Di Rocco M, Panahloo Z, Kuter DJ, Hughes D. Mehta A, et al. Among authors: szer j. Mol Genet Metab. 2017 Nov;122(3):122-129. doi: 10.1016/j.ymgme.2017.08.002. Epub 2017 Aug 4. Mol Genet Metab. 2017. PMID: 28847676 Free article.
Preface to the special issue on Gaucher disease 2017.
Zimran A, Szer J. Zimran A, et al. Among authors: szer j. Blood Cells Mol Dis. 2018 Feb;68:1-2. doi: 10.1016/j.bcmd.2017.08.015. Epub 2017 Sep 8. Blood Cells Mol Dis. 2018. PMID: 28918237 No abstract available.
Recent advances and future challenges in Gaucher disease.
Zimran A, Szer J. Zimran A, et al. Among authors: szer j. Blood Cells Mol Dis. 2018 Feb;68:9-13. doi: 10.1016/j.bcmd.2017.08.016. Epub 2017 Sep 8. Blood Cells Mol Dis. 2018. PMID: 28918238 No abstract available.
436 results