Waisman Center
Advancing knowledge of human development, developmental disabilities, and neurodegenerative diseases.

Long-term velaglucerase alfa treatment in children with Gaucher disease type 1 naïve to enzyme replacement therapy or previously treated with imiglucerase.

Rhead, MD, PHD, W. Long-Term Velaglucerase Alfa Treatment in Children With Gaucher Disease Type 1 naïve to Enzyme Replacement Therapy or Previously Treated With Imiglucerase.

Smith L, Rhead W, Charrow J, Shankar SP, Bavdekar A, Longo N, Mardach R, Harmatz P, Hangartner T, Lee HM, Crombez E, Pastores GM. (2016). Long-term velaglucerase alfa treatment in children with Gaucher disease type 1 naïve to enzyme replacement therapy or previously treated with imiglucerase. Molecular Genetics and Metabolism, 117(2):164-71. doi: 10.1016/j.ymgme.2015.05.012.