Cerdelga (eliglustat) safely and effectively treated three adults with Gaucher disease type 1 (GD1), who switched from long-term use…
Articles by Marisa Wexler MS
People with Gaucher disease are not at increased risk of severe disease if they become infected with SARS-CoV-2, the…
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like…
Children with Gaucher disease who are on enzyme-replacement therapy (ERT) continue to have abnormal levels of immune cells,…
In Spain, a quarter of Gaucher disease patients being given enzyme replacement therapy (ERT) infusions at hospitals experienced dose interruptions due…
A first patient has been treated in a Phase 1/2 trial of AVR-RD-02, Avrobio‘s investigational gene therapy for Gaucher disease…
A case report about two sisters in Brazil with Gaucher type 1 disease (GD1) and severe bone disease represents…
Myelodysplastic syndrome (MDS) is rare in people with Gaucher disease type 1 (GD1) but because it is difficult to…
A new fly model of Gaucher disease may more fully summarize and present the molecular complexities of the disease…