Our products - Giving new hope to patients with key therapeutic solutions
Our products - Giving new hope to patients with key therapeutic solutions

Our Products

Giving new hope to patients with key therapeutic solutions

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Rare diseases

Genzyme, Gustavo, Pompe disease, Brazil

The 6,000 to 8,000 rare diseases are characterized by a broad diversity of disorders and symptoms. 
The patients’ quality of life is affected by the lack or loss of autonomy due to the chronic, progressive and degenerative aspects of the disease.

 

Our Rare Disease business is focused on products (1) for the treatment of rare genetic diseases and other chronic debilitating diseases, including Lysosomal Storage Disorders, or LSDs, a group of metabolic disorders caused by enzyme deficiencies. Our principle rare disease products are enzyme replacement therapies:
 

  • Cerezyme® (imiglucerase) is an enzyme replacement therapy for the treatment of Gaucher disease, an inherited lysosomal storage disease. Nearly 10,000 patients worldwide are estimated to suffer from Gaucher disease.
     
  • Fabrazyme® (agalsidase beta) is an enzyme replacement therapy for the treatment of Fabry disease , an inherited lysosomal storage disease . It is estimated that between 5,000 and 10,000 patients suffer from Fabry disease worldwide.
     
  • Myozyme® (alglucosidase alpha) is an enzyme replacement therapy for the treatment of Pompe disease, an inherited lysosomal storage disease. An estimated 10,000 patients suffer from Pompe disease worldwide.
     
  • Aldurazyme® (laronidase) is an enzyme replacement therapy for the treatment of mucopolysaccharidosis type I (MPS I), a rare genetic disease caused by a deficiency of the enzyme alpha-L-iduronidase, which affects one in 100,000 newborns.


More information on our rare diseases products:

 
 
  1. For more information, please consult the Summaries of Product Characteristics for the products available in your country.
 

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