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

Autor:	Eugen Mengel, Derralynn Hughes, Ana Jovanovic, Sebastiaan J.M. Gaemers, Jyoti Sharma, Tanya Fischer, Raphael Schiffmann, Nigel P. S. Crawford, Pascal Minini
Rok vydání:	2019
Předmět:	Pediatrics medicine.medical_specialty Imiglucerase business.industry Endocrinology Diabetes and Metabolism Safety pharmacology Biochemistry Endocrinology Genetics medicine business Molecular Biology Adult Gaucher disease medicine.drug
Zdroj:	Molecular Genetics and Metabolism. 126:S131
ISSN:	1096-7192
DOI:	10.1016/j.ymgme.2018.12.336
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_________::550df714d0b841d3ccd0c177995ab7b1 https://doi.org/10.1016/j.ymgme.2018.12.336 Zobrazit plný text záznamu Full Text from ScienceDirect