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Content :

Innovating in Rare Diseases

Life is a health journey that for some starts with being born with a rare genetic disorder. There are more than 7,000 rare diseases in the world and 30% of children affected will die before reaching their 5th birthday. Gaucher disease is considered an ultra-rare disease which occurs in approximately 1 in 40,000-60,000 people in the general population, or about 10,000 people globally.

As a health journey partner, Sanofi Genzyme brought to people living with Gaucher disease the first-of-its-kind enzyme replacement therapy given by intravenous infusion to treat this disease in the 1980s. Continuing its commitment to the Gaucher community and through further research and development, Sanofi Genzyme developed an oral therapy for the treatment of certain adults with Gaucher disease type 1, providing another treatment option to support them to continue their lives uninterrupted whilst on treatment.


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Sanofi, Empowering Life.



Updated: December 18, 2017