Work

A phase 3, randomized, double-blind, placebo-controlled, multi-center study to investigate the efficacy and safety of eliglustat in patients with Gaucher disease type 1 (ENGAGE): Results after 9months of treatment

Year: 2013

Type: article

Source: Molecular Genetics and Metabolism

DOI: 10.1016/j.ymgme.2012.11.171

Authors: Pramod Mistry, Elena Lukina, Marie-Françoise Ben Dridi, Dominick Amato, Hagit Baris, Majed Dasouki, Marwan Ghosn, Atul Mehta, Seymour Packman, Gregory Pastores, Milan Petakov, M. Judith Peterschmitt

Institutions: Yale University, National Medical Research Center for Hematology, Hôpital La Rabta, Mount Sinai Hospital, Rabin Medical Center, The University of Kansas Health System, Hôtel-Dieu de France, The Royal Free Hospital, University of California, San Francisco, New York University, Univerzitetski Klinički Centar Srbije

Language: English

Topic: Lysosomal Storage Disorders Research

Subfield: Physiology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: closed

OpenAlex ID: W2319602590

FWCI:

Cites:

Cited by: 1

Related to: 10