Sanofi joins Genzyme in Recognizing International Rare Disease Day
February 28, 2012
This year’s Rare Disease Day marks the first time that Sanofi joins Genzyme in celebrating this important day that was created by the patient organization EURORDIS and involves rare disease organizations globally. The theme of this year is “Solidarity”, emphasizing the importance of collaboration and mutual support in an area where patients are rare and expertise is scarce. The objective is to leverage awareness amongst the general public and decision-makers but also to raise rare diseases as an important public health issue and to bring widespread recognition of increased need for education, research and treatment.
Sanofi and Genzyme’s commitment
Genzyme has pioneered the development and delivery of transformative therapies for patients affected by rare and debilitating diseases for over 30 years. Based in Cambridge (Massachusetts - USA), Genzyme is the global center of excellence for Sanofi in rare diseases, 80% of which are genetic and usually occur in childhood. Many of the diseases for which Genzyme provides treatments have one underlying factor in common: they are lysosomal storage disorders. Patients lack a particular enzyme, which can lead to a wide range of physical, psychosocial and degenerative effects. It causes great suffering and in some cases leads to early death. Thanks to Genzyme’s successful research efforts, patients today benefit from the enzyme replacement treatment for Gaucher Disease, Pompe disease, mucopolysaccharidosis type I, and Fabry disease.
About Rare Diseases
A rare disease means that a relatively small number of people are affected by it. Yet although these diseases are called "rare", there are a large number of patients awaiting solutions: it is estimated that 6-8% of the EU population could be affected by any of the 7,000 known rare diseases, amounting to 25 million people in Europe and the same again in the United States. The proportions appear to be the same around the world.