Roughly 400 million people worldwide are affected by any of the 8,000 known rare diseases; many more people remain undiagnosed. The impact of these conditions ranges from mild to debilitating, and they often affect patients in multiple, complex ways. And because any single disease may affect a dozen people or fewer worldwide, the road to diagnosis and care is often long and uncertain.
Researchers at Ó³»´«Ã½ are accelerating progress in the study and treatment of rare diseases in three main areas.