About the Authors

John Marshall

john.marshall@genzyme.com

Affiliation Applied Discovery Research, Genzyme Corporation, Framingham, Massachusetts, United States of America

Karen M. Ashe

Affiliation Applied Discovery Research, Genzyme Corporation, Framingham, Massachusetts, United States of America

Dinesh Bangari

Affiliation Applied Discovery Research, Genzyme Corporation, Framingham, Massachusetts, United States of America

KerryAnne McEachern

Affiliation Applied Discovery Research, Genzyme Corporation, Framingham, Massachusetts, United States of America

Wei-Lien Chuang

Affiliation Applied Discovery Research, Genzyme Corporation, Framingham, Massachusetts, United States of America

Joshua Pacheco

Affiliation Applied Discovery Research, Genzyme Corporation, Framingham, Massachusetts, United States of America

Diane P. Copeland

Affiliation Applied Discovery Research, Genzyme Corporation, Framingham, Massachusetts, United States of America

Robert J. Desnick

Affiliation Genetics and Genomic Sciences, Mount Sinai School of Medicine, New York, New York, United States of America

James A. Shayman

Affiliation Internal Medicine – Nephrology, Genzyme Corporation, University of Michigan, Ann Arbor, Michigan, United States of America

Ronald K. Scheule

Affiliation Applied Discovery Research, Genzyme Corporation, Framingham, Massachusetts, United States of America

Seng H. Cheng

Affiliation Applied Discovery Research, Genzyme Corporation, Framingham, Massachusetts, United States of America

Competing Interests

The authors have read the journal's policy and have the following conflicts: John Marshall, Karen Ashe, Dinesh Bangari, Kerry Anne McEachern, Wei-Lien Chuang, Joshua Pacheco, Diane Copeland, Ronald Scheule, and Seng Cheng are all employees of and shareholders in Genzyme Corporation. James Shayman is an employee of the University of Michigan and co-inventor of Genz-112638 which is licensed to Genzyme by the University of Michigan. Genzyme funded all the studies and is developing Eliglustat tartrate for treatment of type 1 Gaucher disease. Patents regarding this approach have been filed. This does not alter the authors' adherence to all the PLoS ONE policies on sharing data and materials.

Author Contributions

Conceived and designed the experiments: JM KMA DB KM DPC JAS RKS SHC. Performed the experiments: JM KMA DB KM WC JP. Analyzed the data: JM KMA DB WC JP DPC RJD JAS RKS SHC. Contributed reagents/materials/analysis tools: JM DB WC DPC RJD JAS. Wrote the paper: JM RKS SHC.