Randomized, controlled trial of miglustat in Gaucher's disease type 3

2008232 citationsJournal Articlebronze Open Access

Authors

Raphael Schiffmann

Edmond J. FitzGibbon · National Institutes of Health

Chris Harris · University of Plymouth

Catherine DeVile · University College London

Elin Haf Davies · Great Ormond Street Hospital

Larry A. Abel · Australian College of Optometry

Ivo N. van Schaik · University of Amsterdam

Abstract

Miglustat does not appear to have significant benefits on the neurological manifestations of GD3. However, miglustat may have positive effects on systemic disease (pulmonary function and chitotriosidase activity) in addition to ERT in patients with GD3.

Topics & Keywords

Lysosomal Storage Disorders Research Carbohydrate Chemistry and Synthesis Cellular transport and secretion

UN Sustainable Development Goals

Good health and well-being

Publication Details

Published in: Annals of Neurology

Volume 64, Issue 5, pp. 514-522

DOI: 10.1002/ana.21491

Field-Weighted Citation Impact: 9.16