Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Oral Venglustat in Healthy Volunteers

Venglustat in adult Gaucher disease type 3: Preliminary safety, pharmacology, and exploratory efficacy from a phase 2 trial in combination with imiglucerase (LEAP)

(2019) Raphael Schiffmann et al.   MOLECULAR GENETICS AND METABOLISM

Safety, tolerability and pharmacokinetics of oral venglustat in Parkinson disease patients with a GBA mutation

(2019) M. Judith Peterschmitt et al.   MOLECULAR GENETICS AND METABOLISM

Genetics and Therapies for GM2 Gangliosidosis

(2018) Maria Begona Cachon-Gonzalez et al.   CURRENT GENE THERAPY

The role of glucocerebrosidase in Parkinson disease pathogenesis

(2018) Matthew E. Gegg et al.   FEBS Journal

Targeting Glucosylceramide Synthesis in the Treatment of Rare and Common Renal Disease

(2018) James A. Shayman   SEMINARS IN NEPHROLOGY

A Review of Gaucher Disease Pathophysiology, Clinical Presentation and Treatments

(2017) Jérôme Stirnemann et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Sphingolipids and their metabolism in physiology and disease

(2017) Yusuf A. Hannun et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

The Complicated Relationship between Gaucher Disease and Parkinsonism: Insights from a Rare Disease

(2017) Elma Aflaki et al.   NEURON

Glucosylceramide synthase inhibition alleviates aberrations in synucleinopathy models

(2017) S. Pablo Sardi et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Perspective: 4β-hydroxycholesterol as an emerging endogenous biomarker of hepatic CYP3A

(2016) Jialin Mao et al.   DRUG METABOLISM REVIEWS

CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease

(2016) John Marshall et al.   MOLECULAR THERAPY

Unknown

(2015) Karen Ashe et al.   MOLECULAR MEDICINE

A novel approach for quantitation of glucosylceramide in human dried blood spot using LC–MS/MS

(2015) Allena Ji Ji et al.   Bioanalysis

Validation of 4β-hydroxycholesterol and evaluation of other endogenous biomarkers for the assessment of CYP3A activity in healthy subjects

(2014) Sreeneeranj Kasichayanula et al.   BRITISH JOURNAL OF CLINICAL PHARMACOLOGY

Loss of GM3 synthase gene, but not sphingosine kinase 1, is protective against murine nephronophthisis-related polycystic kidney disease

(2012) Thomas A. Natoli et al.   HUMAN MOLECULAR GENETICS

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

Inhibition of glucosylceramide accumulation results in effective blockade of polycystic kidney disease in mouse models

(2010) Thomas A Natoli et al.   NATURE MEDICINE