Should eliglustat be first line therapy for patients with type 1 Gaucher disease? Definitions of safety and efficacy

Blood Cells Mol Dis. 2018 Feb:68:14-16. doi: 10.1016/j.bcmd.2017.09.003. Epub 2017 Sep 14.

Authors

Ari Zimran¹, Jack Goldblatt², Jeff Szer³

Affiliations

¹ Gaucher Clinic, Shaare Zedek Medical Center, 12 Bayit Street, Jerusalem 91031, Israel.
² Genetics Services of Western Australia, 374 Bagot Rd, Subiaco, WA 6008, Australia.
³ Department of Clinical Haematology, Royal Melbourne Hospital and Victorian Comprehensive Cancer Centre, 300 Grattan St, Parkville, VIC 3050, Australia.. Electronic address: jeff.szer@mh.org.au.

PMID: 28935503
DOI: 10.1016/j.bcmd.2017.09.003

No abstract available

Keywords: Clinical trial design; Eliglustat; Enzyme replacement; Gaucher; Substrate reduction.

Publication types

Review

MeSH terms

Clinical Trials, Phase III as Topic
Enzyme Inhibitors / adverse effects*
Enzyme Inhibitors / therapeutic use*
Enzyme Replacement Therapy
Gaucher Disease / drug therapy*
Humans
Pyrrolidines / adverse effects*
Pyrrolidines / therapeutic use*
Treatment Outcome

Substances

Enzyme Inhibitors
Pyrrolidines
eliglustat