They're called "rare diseases." The term suggests they affect a handful of individuals, isolated cases, and medical exceptions. However, this impression is misleading. In France, more than 3 million people are affected by a rare disease. Worldwide, more than 300...
Sanfilippo syndrome, also known as Mucopolysaccharidosis Type III (MPS III), is a rare genetic disorder that affects the brain. It causes a progressive decline in mental and physical abilities, leading to a loss of cognitive and motor functions. Symptoms typically...