- Article
- Source: Campus Sanofi
- 23 Oct 2023
Our commitment to rare disease
This is intended for HCPs practising in Great Britain (England, Scotland, Wales) only.
Watch the video below to find out how our 30-year commitment to rare disease1 means we always lead with science, and put patients, their families, and the community at the centre of everything we do.
Why Nexviadyme was developed
From the challenges of cell delivery, to optimising targeting,2,4 explore why Sanofi developed Nexviadyme®.
How Nexviadyme® was designed
Watch how we used preclinical mice models of Pompe to design Nexviadyme®, and improve maintenance of muscle strength compared to Myozyme®.3,5
Why Nexviadyme was developed
From the challenges of cell delivery, to optimising targeting,2,4 explore why Sanofi developed Nexviadyme®.
How Nexviadyme® was designed
Watch how we used preclinical mice models of Pompe to design Nexviadyme®, and improve maintenance of muscle strength compared to Myozyme®.3,5
References
- Mistry, P.K. et al. Rare lysosomal disease registries: lessons learned over three decades of real-world evidence. Orphanet Journal of Rare Diseases. 2022;17:362.
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Chien YH, et al. Long-term prognosis of patients with infantile-onset Pompe disease diagnosed by newborn screening and treated since birth. Journal of Pediatrics. 2015 Apr;166(4):985-91.e1-2.
- Sanofi. Nexviadyme (avalglucosidase alfa). Summary of Product Characteristics. 2023. Available at: https://www.medicines.org.uk/emc/product/14562/smpc#gref.
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Zhu Y, et al. Carbohydrate-remodelled acid alpha-glucosidase with higher affinity for the cation-independent mannose 6-phosphate receptor demonstrates improved delivery to muscles of Pompe mice. The Biochemical Journal. 2005 Aug 1;389(Pt 3):619-628.
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Zhu Y, et al. Glycoengineered acid alpha-glucosidase with improved efficacy at correcting the metabolic aberrations and motor function deficits in a mouse model of Pompe disease. Molecular Therapy. 2009 Jun;17(6):954-63.
MAT-XU-2302328 (v3.0) Date of preparation: December 2023