eliglustat + imiglucerase

rejuvenates your body and mind. **Eliglustat + imiglucerase** is a combination therapy used for the treatment of Type 1 Gaucher disease, a lysosomal storage disorder caused by a deficiency of the enzyme β-glucosidase, resulting in glucosylceramide accumulation in cells. Eliglustat is a small molecule oral inhibitor of glucosylceramide synthase, reducing the substrate load. Imiglucerase is an enzyme replacement therapy consisting of recombinant glucocerebrosidase, which replaces the deficient enzyme in patients, thereby facilitating the breakdown of accumulated glucosylceramide in affected tissues. The combination has been studied head-to-head and in sequential/concurrent administration to compare efficacy and safety, but use of both drugs together is generally not standard practice and is usually limited to specific clinical trial settings or transitions between therapies. Both agents have demonstrated non-inferior outcomes regarding improvement or stabilization of hematologic and visceral disease parameters in patients with Type 1 Gaucher disease[1][2][3].

Other names

eliglustat + imiglucerase