D2I2.
genetic

Sanfilippo syndrome

Sanfilippo , also known as type III, is a rare lifelong disease that mainly affects the brain and spinal cord. It is caused by a problem with how the body breaks down certain large sugar molecules called . In children with this condition, these sugar molecules build up in the body and eventually lead to damage of the central nervous system and other organ systems.

Underlined words are explained — tap any of them.

Symptoms — what it feels like

  • ·Progressive intellectual disability; ; ; loss of mobility

Causes — why it happens

  • ·Inherited

How it's found

  • ·MPS urine screen (initial test), confirmed by blood test

Treatment

  • ·There is no cure; only symptom management

Outlook

  • ·Lifespan is reduced; most patients survive until the early teenage years, but some may reach their 30s
Plain-language summary adapted from Wikipedia. Not medical advice.