The effects of Sanfilippo syndrome in small children are so devastating that the rare disease is often known as "childhood dementia."
About one in 70,000 newborns inherit this disorder. The affected children look normal at birth, but between ages one and six they begin to develop a host of symptoms, including severe developmental delays, seizure-like episodes, and losses in cognition, vision and hearing. These symptoms are often accompanied by chronic pain and general emotional distress. After a number of years, patients become unresponsive. Without exception, Sanfilippo leads to an early death.
