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Page 1
The risk of Parkinson's disease in type 1 Gaucher disease.
Bultron G, Kacena K, Pearson D, Boxer M, Yang R, Sathe S, Pastores G, Mistry PK. Bultron G, et al. Among authors: kacena k. J Inherit Metab Dis. 2010 Apr;33(2):167-73. doi: 10.1007/s10545-010-9055-0. Epub 2010 Feb 23. J Inherit Metab Dis. 2010. PMID: 20177787 Free PMC article.
Glucosylsphingosine is a key biomarker of Gaucher disease.
Murugesan V, Chuang WL, Liu J, Lischuk A, Kacena K, Lin H, Pastores GM, Yang R, Keutzer J, Zhang K, Mistry PK. Murugesan V, et al. Among authors: kacena k. Am J Hematol. 2016 Nov;91(11):1082-1089. doi: 10.1002/ajh.24491. Epub 2016 Aug 8. Am J Hematol. 2016. PMID: 27441734 Free PMC article.
Life expectancy in Gaucher disease type 1.
Weinreb NJ, Deegan P, Kacena KA, Mistry P, Pastores GM, Velentgas P, vom Dahl S. Weinreb NJ, et al. Am J Hematol. 2008 Dec;83(12):896-900. doi: 10.1002/ajh.21305. Am J Hematol. 2008. PMID: 18980271 Free PMC article.
Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study.
Pena LDM, Barohn RJ, Byrne BJ, Desnuelle C, Goker-Alpan O, Ladha S, Laforêt P, Mengel KE, Pestronk A, Pouget J, Schoser B, Straub V, Trivedi J, Van Damme P, Vissing J, Young P, Kacena K, Shafi R, Thurberg BL, Culm-Merdek K, van der Ploeg AT; NEO1 Investigator Group. Pena LDM, et al. Among authors: kacena k. Neuromuscul Disord. 2019 Mar;29(3):167-186. doi: 10.1016/j.nmd.2018.12.004. Epub 2018 Dec 17. Neuromuscul Disord. 2019. PMID: 30770310 Free article. Clinical Trial.
23 results