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Gaucher disease is an inherited metabolic disease characterized by β-glucocerebrosidase deficiency and commonly treated with enzyme replacement therapy (ERT). The efficacy of ERT with velaglucerase alfa was assessed based on...

The FDA on 1st May 2012 approved Elelyso (taliglucerase alfa) from Pfizer for long-term enzyme replacement therapy to treat a...

This retrospective, exploratory analysis of data from phase 3 clinical trials of velaglucerase alfa in patients with type 1 GD evaluated the potential of lyso-Gb1 as a specific and sensitive biomarker for GD.

Following the treatment of the first Gaucher disease patient with enzyme replacement therapy (ERT), it was clear that ERT had the potential to be transformative with dramatic improvements...

Purpose: In Gaucher disease, diminished activity of the lysosomal enzyme, acid β-glucosidase, leads to accumulation of glucosylceramides and related substrates, primarily in the spleen, liver, and bone marrow.

Sanofi and its subsidiary Genzyme announced that the European Commission (EC) has granted marketing authorization for Cerdelga (eliglustat) capsules, a...

Sanofi and its subsidiary Genzyme, announced the publication of results from the ENGAGE registration study evaluating Cerdelga (eliglustat) in treatment-naïve...

In the phase 3 trial of eliglustat in patients with Gaucher disease type 1 already stabilized with enzyme therapy (ENCORE), at one year, eliglustat was non-inferior to imiglucerase enzyme therapy in maintaining stable platelet counts ...

Pivotal study data published in The New England Journal of Medicine (NEJM) continues to highlight the efficacy, safety, and pharmacokinetic profile of efanesoctocog alfa, an investigational treatment for hemophilia A