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

Publication date: February 2019Source: Molecular Genetics and Metabolism, Volume 126, Issue 2Author(s): Raphael Schiffmann, Eugen Mengel, Nigel Crawford, Sebastiaan Gaemers, Derralynn Hughes, Ana Jovanovic, Pascal Minini, Jyoti Sharma, Tanya Fischer
Source: Molecular Genetics and Metabolism - Category: Genetics & Stem Cells Source Type: research