9e. Unmet Needs in the Treatment of GD in the Era of Macrophage-Targeted Enzyme Therapy


Rosenbloom BE, et al. Crit Rev Oncog. 2013;18:163-75; Desnick RJ, et al. Annu Rev Genomics Hum Genet. 2012;13:307-35;
Vitner EB, et al. Brain. 2012;135:1724-35; Cox TM. Biologics. 2010;4:299-313; Mehta A. Acta Paediatr. Suppl. 2008;97:83-7.

These are some of the unmet needs in the treatment of Gaucher Disease. Focussing on the pediatric population, the biggest concern is that we have no therapies for type 2 and type 3 Gaucher Disease, including type 3 Gaucher Disease that has the cardiac phenotype, and some of the severe destructive skeletal manifestations, and so in the future the hope is that we will have therapies that will allow us to look after children affected by the devastating forms of Gaucher Disease that are listed here.







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