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ERA 2022 | May 19, 2022 - May 22, 2022

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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  • Alport

    Alport syndrome is a rare, inherited disorder of basement membranes caused by pathogenic variants in type IV collagen genes (COL4A3, COL4A4, and COL4A5) leading to progressive chronic kidney disease, hearing loss, and eye abnormalities in most affected individuals.