Making the voice of rare diseases heard

Rare diseases
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Defined on the basis of their very low prevalence – affecting fewer than 200,000 Americans at any given timerare diseases are very diverse in character, but nevertheless affect a very large number of people. There are approximately 7,000 rare diseases, affecting around 30 million patients in the United States. 80% of rare diseases have identified genetic origins, and half emerge only in adulthood. Despite the fact that the majority of them are serious and debilitating, most rare diseases remain little understood in terms of their science.

 

Among them, Pompe disease affects an estimated 50,000 people worldwide.  It is a progressive, debilitating and often fatal neuromuscular disease caused by a genetic deficiency. Patients often lose their ability to walk and require wheelchairs to assist with mobility. They also often experience difficulty breathing and may require mechanical ventilation to breathe.

 

In order to attract the attention of the general public and the relevant authorities, non-profit organizations and healthcare stakeholders launched an awareness appeal on Monday, February 29 to coincide with International Rare Disease Day.

All the organizers and contributors rallied around this year’s slogan ‘Join us in making the voice of rare diseases heard’. As for Sanofi Genzyme, the company decided to championing Patient Voice through Artwork reports Richard Peters, Senior Vice President and Global Rare Diseases Franchise Head at Sanofi Genzyme on Speaking of Sanofi’s blog.

 

For a decade, Genzyme uses its Expression of Hope art program to raise awareness of lysosomal storage disorders, a group of diseases for which the laboratory has discovered and developed a number of different treatments. In recognition of the 10 year anniversary of the program, the company created a commemorative eBook and a series of short video interviews with several of the artists.

 

“This collection is dedicated to all those who have been touched by lysosomal storage disorders and who inspired us and the entire world through their perseverance, candor, courage and hope for the future” says Richard Peters.

 

With 35 years of commitment to rare diseases, Sanofi Genzyme is a pioneer in research in this area.

 

read the article on Speaking of Sanofi

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