Presentation
Safety and Efficacy of Long-Term Velaglucerase Alfa Therapy in Treatment-Naïve Adults with Type 1 Gaucher Disease: Results from Phase III Trials
Molecular Genetics and Metabolism
Document Type
Abstract
Publication Date
2-1-2014
Disciplines
Abstract
A subgroup analysis of the safety and efficacy of velaglucerase alfa in adult patients (age ≥ 18 years) with type I Gaucher disease (GD1) who were treatment-naïve at advent of trial participation is presented.
DOI
10.1016/j.ymgme.2013.12.097
Citation Information
Pilar Giraldo, Ari Zimran, Atul Mehta, Derralynn A. Hughes, et al.. "Safety and Efficacy of Long-Term Velaglucerase Alfa Therapy in Treatment-Naïve Adults with Type 1 Gaucher Disease: Results from Phase III Trials" Molecular Genetics and Metabolism Vol. 111 Iss. 2 (2014) p. S47 ISSN: 10967192 Available at: http://works.bepress.com/thomas_hangartner/127/
Presented at the Lysosomal Disease Network WORLD Symposium, San Diego, CA, February 11-13, 2014.