NextBillion - An Initiative of the William Davidson Institute at the University of Michigan

Friday, August 30, 2013

Rare diseases might be called that, but they affect 3.5 million people in the UK alone. Most have no cure. Funding research into these diseases is challenging. But, when it does take place, it often leads to the discovery of potential treatments for chronic conditions from which many more people suffer.

Take statins: developed initially for a rare disease called familial hypercholesterolemia, they are now used widely to treat high cholesterol, which is one of the main risk factors for heart disease. Or bisphosphonates: a class of drugs developed initially for a rare disease called hypophosphatasia, they are now used extensively to treat common bone disorders such as osteoporosis.

Recognising the power of studying these rare diseases, Ashoka fellow Nick Sireau launched Findacure. Run as a social enterprise, the organisation aims to raise awareness, fund research, unlock new insights and discover potential treatments. Rather than rare diseases, they speak of “fundamental diseases”.

“The word rare has connotations of not being important, or being quirky, not affecting many people,” says Sireau. “People don’t feel affected by it. If it’s rare, why care? They are fundamental diseases: extreme and exceptional diseases that advance our understanding of medicine and help us discover potential new treatments.”