Functioning brain cells need a functioning system for picking up the trash and sorting the recycling. But when the cellular ...

Analysis from continued follow-up of Phase 1/2 clinical trial data in Hunter syndrome (MPS II) reinforces potential for tividenofusp alfa ...

Chiesi’s abstract presentations will focus on clinical insights and patient-reported outcomes in Fabry disease and ...

Sanofi’s venglustat met all primary endpoints in a phase 3 study of type 3 Gaucher diseaseIn the LEAP2MONO phase 3 study, venglustat, dosed ...

Knowing which lysosomal proteins are enriched in which brain cell types will help better understand contribution to neurological disorders.

JCR Pharmaceuticals Co., Ltd. (TSE 4552; "JCR"), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, today announced the presentation of four ...

Together with colleagues from Stanford University, USA, researchers at the Leibniz Institute on Aging (FLI) have, for the first time, created a comprehensive cell type-specific atlas of lysosomes in ...

Sanofi will take venglustat to regulators for Gaucher disease but an application for Fabry disease is less clear after the failure of a Phase III trial.

LONDON--(BUSINESS WIRE)--Technavio’s latest market research report on the global lysosomal storage diseases (LSDs) market provides an analysis of the most important trends expected to impact the ...