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EHA 2024 | Jun 13, 2024 - Jun 16, 2024

Rare Diseases

Lysosomal storage disorders (LSDs)—a group of rare genetic conditions caused by enzyme deficiencies—are one of Sanofi’s proudest business cornerstones and the medical area for which it is most well-known. Focusing on LSDs and other uncommon and underserved medical conditions, Sanofi’s Rare Disease franchise is committed to empowering the lives of patients with rare diseases by offering sustainable, transformative healthcare options resulting in Better Care for Rare.

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  • Gaucher

    Gaucher disease is an autosomal recessive disorder caused by a deficiency in glucocerebrosidase (GBA) enzyme activity. Deficiency or absence of the enzyme leads to buildup of glycosylceramide (GL-1) and glucosylsphingosine (lyso-GL-1). Three clinical types are delineated by the absence (type 1) or presence (types 2 and 3) of primary CNS involvement. Type 3 disease is a chronic neuronopathic disorder with wide-ranging effects and diverse neurological manifestations.