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ASN 2024 | Oct 23, 2024 - Oct 27, 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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  • Pipeline

  • Fabry

    Fabry disease is an X-linked, multi-systemic, genetic disorder of glycosphingolipid metabolism. Clinical manifestations are due to an absence or deficiency of lysosomal α-Galactosidase A caused by pathogenic variants in the GLA gene.