A novel tool for mapping disease severity and outcomes in patients with Gaucher disease utilising the therapeutic goals

作者:Weinreb Neal; Belmatoug Nadia; Bembi Bruno; Bodamer Olaf; Cappellini Maria Domenica; Deegan Patrick B; Elstein Deborah; Faran Gil; Gilrado Pilar; Hughes Derralynn A; Mengel Eugen; Pastores Gregory M; Zimran Ari
来源:7th Annual World Symposium of the Lysosomal-Disease-Network, 2011-02-16 to 2011-02-18.
DOI:10.1016/j.ymgme.2010.11.154