Ethylenedioxy-PIP2 Oxalate Reduces Ganglioside Storage in Juvenile Sandhoff Disease Mice

Eliglustat tartrate

(2014) J.A. Shayman   DRUGS OF THE FUTURE

Therapeutic Effects of Stem Cells and Substrate Reduction in Juvenile Sandhoff Mice

(2012) J. R. Arthur et al.   NEUROCHEMICAL RESEARCH

Substrate reduction therapy with miglustat for type 1 Gaucher disease: A retrospective analysis from a single institution

(2012) Maciej Machaczka et al.   UPSALA JOURNAL OF MEDICAL SCIENCES

Property-based design of a glucosylceramide synthase inhibitor that reduces glucosylceramide in the brain

(2011) Scott D. Larsen et al.   JOURNAL OF LIPID RESEARCH

Iminosugar-Based Inhibitors of Glucosylceramide Synthase Increase Brain Glycosphingolipids and Survival in a Mouse Model of Sandhoff Disease

(2011) Karen M. Ashe et al.   PLoS One

A phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1

(2010) E. Lukina et al.   BLOOD

Improvement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (Genz-112638) treatment: 2-year results of a phase 2 study

(2010) E. Lukina et al.   BLOOD

Improved management of lysosomal glucosylceramide levels in a mouse model of type 1 Gaucher disease using enzyme and substrate reduction therapy

(2010) John Marshall et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Substrate Reduction Augments the Efficacy of Enzyme Therapy in a Mouse Model of Fabry Disease

(2010) John Marshall et al.   PLoS One

Miglustat (Zavesca®) in type 1 Gaucher disease: 5-year results of a post-authorisation safety surveillance programme

(2009) Carla E. M. Hollak et al.   PHARMACOEPIDEMIOLOGY AND DRUG SAFETY

Peripheral blood mononuclear cell infiltration and neuroinflammation in the HexB−/− mouse model of neurodegeneration

(2008) Stephanos Kyrkanides et al.   JOURNAL OF NEUROIMMUNOLOGY

Comparative Analysis of Brain Lipids in Mice, Cats, and Humans with Sandhoff Disease

(2008) Rena C. Baek et al.   LIPIDS

Beneficial effects of substrate reduction therapy in a mouse model of GM1 gangliosidosis

(2008) Elena Elliot-Smith et al.   MOLECULAR GENETICS AND METABOLISM

N-butyldeoxygalactonojirimycin reduces brain ganglioside and GM2 content in neonatal Sandhoff disease mice

(2007) Rena C. Baek et al.   NEUROCHEMISTRY INTERNATIONAL