What is Gaucher Disease?
Gaucher disease, also known as glucocerebrosidase, or GCD, deficiency, is a rare genetic autosomal recessive disorder and one of the most common Lysosomal Storage Disorders, or LSDs, in the world. It is one of a group of disorders that affect specific enzymes that normally break down materials for reuse in the cells, resulting in toxic accumulation of material. Gaucher disease occurs when a lipid called glucosylceramide accumulates in the bone marrow, lungs, spleen, liver, and sometimes the brain. Symptoms can include fatigue, anemia, easy bruising and bleeding, severe bone pain and easily broken bones, and distended stomach due to an enlarged spleen and thrombocytopenia. It is present in approximately 1 in 20,000 live births.
ABOUT ELELYSO®
Elelyso® (taliglucerase alfa) is an Enzyme Replacement Therapy (ERT), replacing the deficient glucocerebrosidase enzyme with a recombinant form of the protein. Taliglucerase alfa is the company’s first approved drug product produced through ProCellEx® and the first plant cell-based recombinant therapeutic protein approved by the FDA or any other major regulatory authority.
For more information please see: www.elelyso.com.
