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ISPOR EU 2026 | Nov 8, 2026 - Nov 11, 2026

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 disease

    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.

  • Alpha-1 antitrypsin deficiency

  • CIDP

    Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare, acquired autoimmune disease of the peripheral nerves and nerve roots. The immune-mediated loss of myelin sheaths that occurs in CIDP classically leads to proximal and distal muscle weakness and numbness. Characterized as a relapsing or progressive disease, the disorder ultimately results in disability accumulation. Diagnosis of CIDP is challenging due to its rarity, heterogeneous presentation, and identification by exclusion.

  • Immunoglobulin-G4-related disease

  • FSHD