About the Authors

Cristin D. Davidson

Affiliation Dominick P. Purpura Department of Neuroscience, Rose F. Kennedy Center for Research in Mental Retardation and Human Development, Albert Einstein College of Medicine, Bronx, New York, United States of America

Nafeeza F. Ali

Affiliation Dominick P. Purpura Department of Neuroscience, Rose F. Kennedy Center for Research in Mental Retardation and Human Development, Albert Einstein College of Medicine, Bronx, New York, United States of America

Matthew C. Micsenyi

Affiliation Dominick P. Purpura Department of Neuroscience, Rose F. Kennedy Center for Research in Mental Retardation and Human Development, Albert Einstein College of Medicine, Bronx, New York, United States of America

Gloria Stephney

Affiliation Dominick P. Purpura Department of Neuroscience, Rose F. Kennedy Center for Research in Mental Retardation and Human Development, Albert Einstein College of Medicine, Bronx, New York, United States of America

Sophie Renault

Affiliation Institut National de la Sante et de la Recherche Medicale Unit 820, Laennec Medical School and Lyon-1 University, Lyon, France

Kostantin Dobrenis

Affiliation Dominick P. Purpura Department of Neuroscience, Rose F. Kennedy Center for Research in Mental Retardation and Human Development, Albert Einstein College of Medicine, Bronx, New York, United States of America

Daniel S. Ory

Affiliations Department of Medicine, Washington University School of Medicine, St. Louis, Missouri, United States of America, Department of Cell Biology and Physiology, Washington University School of Medicine, St. Louis, Missouri, United States of America

Marie T. Vanier

Affiliation Institut National de la Sante et de la Recherche Medicale Unit 820, Laennec Medical School and Lyon-1 University, Lyon, France

Steven U. Walkley

walkley@aecom.yu.edu

Affiliation Dominick P. Purpura Department of Neuroscience, Rose F. Kennedy Center for Research in Mental Retardation and Human Development, Albert Einstein College of Medicine, Bronx, New York, United States of America

Competing Interests

SUW is an author on a patent for the use of miglustat for the treatment of Niemann-Pick C disease. MTV has been a paid and non-paid consultant for Actelion, which markets miglustat as treatment for Niemann-Pick C disease.

Author Contributions

Conceived and designed the experiments: SUW. Performed the experiments: CDD NFA MCM GS SR MTV. Analyzed the data: CDD KD MTV. Contributed reagents/materials/analysis tools: MTV SUW. Wrote the paper: CDD SUW. Contributed to writing of autophagy sections and editing of paper: MCM. Assisted with microscopy, data analysis, and editing of paper: KD. Major collaborator who contributed importantly to discussion section of paper: DSO. Contributed to writing sections dealing with biochemical anaylsis of gangliosides and to editing the paper: MTV.