- Article
- Source: Campus Sanofi
- 23 Oct 2023
Our commitment to rare disease
![](https://pro.campus.sanofi/.imaging/mte/portal/3840/dam/Portal/UK/Articles/hero-our-commitment.jpg/jcr:content/hero%20our%20commitment.jpg)
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.
![](https://pro.campus.sanofi/.imaging/mte/portal/3840/dam/Portal/UK/Articles/card-2-violet.jpg/jcr:content/card%202%20violet.jpg)
Why Nexviadyme was developed
From the challenges of cell delivery, to optimising targeting,2,4 explore why Sanofi developed Nexviadyme®.
![](https://pro.campus.sanofi/.imaging/mte/portal/3840/dam/Portal/UK/Products/big-card-molecule-green.jpg0/jcr:content/big%20card%20molecule%20green.jpg)
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
![](https://pro.campus.sanofi/.imaging/mte/portal/3840/dam/Portal/UK/Articles/card-2-violet.jpg/jcr:content/card%202%20violet.jpg)
Why Nexviadyme was developed
From the challenges of cell delivery, to optimising targeting,2,4 explore why Sanofi developed Nexviadyme®.
![](https://pro.campus.sanofi/.imaging/mte/portal/3840/dam/Portal/UK/Products/big-card-molecule-green.jpg0/jcr:content/big%20card%20molecule%20green.jpg)
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