Liver fibrosis is a common condition in patients with Gaucher disease type 1 (GD1) and can be predicted by…
Articles by Jose Marques Lopes PhD
Shortening the duration of VPRIV (velaglucerase alfa) infusions from 60 to 10 minutes does not affect the safety or effectiveness of…
Patients with Gaucher disease type 1 (GD1) may exhibit neurological symptoms at a young age, requiring the use of…
A new study reported the incidence rates of Gaucher disease and three other lysosomal storage disorders in the…
Three new mutations in three of four babies with Gaucher disease type 2 were reported in a Turkish study, further…
Centogene and Orphazyme will collaborate in a Phase 2 clinical trial of arimoclomol, an experimental therapy for …
New Zealand’s Pharmaceutical Management Agency (PHARMAC) is requesting feedback on a proposed change in the enzyme replacement therapy…
Testing dried blood spots is an easy, inexpensive, and effective way for the early diagnosis of Gaucher disease (GD)…
Sanofi Genzyme is launching a Phase 3 clinical trial testing Cerdelga (eliglustat) with or without Cerezyme (imiglucerase) in children…
An experimental, non-invasive stem cell-based therapy led to improved motor function, prolonged lifespan, and reduced neurodegeneration in a mouse model…