Title

Safety and Efficacy of Long-Term Velaglucerase Alfa Therapy in Treatment-Naïve Adults with Type 1 Gaucher Disease: Results from Phase III Trials

Document Type

Abstract

Publication Date

2-2014

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.

Comments

Presented at the Lysosomal Disease Network WORLD Symposium, San Diego, CA, February 11-13, 2014.

DOI

10.1016/j.ymgme.2013.12.097