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ISPOR-US 2025 | May 13, 2025 - May 16, 2025

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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  • GM2 Gangliosidoses

    GM2 gangliosidoses comprises rare, autosomal recessive, neurodegenerative lysosomal storage disorders, primarily Tay-Sachs disease and Sandhoff disease. Pathogenic variants lead to a deficiency in the activity of the enzyme (β-hexosaminidase) responsible for catabolizing GM2 ganglioside. This causes lysosomal accumulation of its substrate in brain and nerve cells.