Eliglustat: A Review in Gaucher Disease Type 1

Effect of Oral Eliglustat on Splenomegaly in Patients With Gaucher Disease Type 1

(2015) Pramod K. Mistry et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Eliglustat compared with imiglucerase in patients with Gaucher's disease type 1 stabilised on enzyme replacement therapy: a phase 3, randomised, open-label, non-inferiority trial

(2015) Timothy M Cox et al.   LANCET

ENCORE, a randomized, controlled, open-label non-inferiority study comparing eliglustat to imiglucerase in Gaucher disease type 1 patients stabilized on enzyme replacement therapy: 24-month results

(2015) Timothy M. Cox et al.   MOLECULAR GENETICS AND METABOLISM

ENGAGE — A phase 3, randomized, double-blind, placebo-controlled, multi-center study to investigate the efficacy and safety of eliglustat in adults with Gaucher disease type 1: Results after 18months

(2015) Pramod Mistry et al.   MOLECULAR GENETICS AND METABOLISM

Clinical response to eliglustat in treatment-naive patients with Gaucher disease type 1: Post-hoc comparison to imiglucerase in a real-world setting

(2015) M. Judith Peterschmitt et al.   MOLECULAR GENETICS AND METABOLISM

Eliglustat, an investigational oral therapy for Gaucher disease type 1: Phase 2 trial results after 4years of treatment

(2014) Elena Lukina et al.   BLOOD CELLS MOLECULES AND DISEASES

Eliglustat tartrate

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

The development and use of small molecule inhibitors of glycosphingolipid metabolism for lysosomal storage diseases

(2014) James A. Shayman et al.   JOURNAL OF LIPID RESEARCH

Eliglustat safety profile based on a pooled analysis of data from four trials in Gaucher disease type I

(2014) Leorah Ross et al.   MOLECULAR GENETICS AND METABOLISM

Skeletal improvement in patients with Gaucher disease type 1: a phase 2 trial of oral eliglustat

(2014) Ravi S. Kamath et al.   SKELETAL RADIOLOGY

Clinical Pharmacogenetics Implementation Consortium Guideline for CYP2D6 and CYP2C19 Genotypes and Dosing of Tricyclic Antidepressants

(2013) J K Hicks et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Lysosomal storage diseases: Diagnostic confirmation and management of presymptomatic individuals

(2011) Raymond Y Wang et al.   GENETICS IN MEDICINE

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

Safety, Tolerability, and Pharmacokinetics of Eliglustat Tartrate (Genz-112638) After Single Doses, Multiple Doses, and Food in Healthy Volunteers

(2010) M. Judith Peterschmitt et al.   JOURNAL OF CLINICAL PHARMACOLOGY

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

Recommendations on Diagnosis, Treatment, and Monitoring for Gaucher Disease

(2009) Ana Maria Martins et al.   JOURNAL OF PEDIATRICS