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

Laurie Smith*, William Rhead, Joel Charrow, Suma P. Shankar, Ashish Bavdekar, Nicola Longo, Rebecca Mardach, Paul Harmatz, Thomas Hangartner, Hak Myung Lee, Eric Crombez, Gregory M. Pastores

*Corresponding author for this work

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24 Scopus citations

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Pharmacology, Toxicology and Pharmaceutical Science