Prognosis of Sanfilippo

At present, MPS III is terminal.

At this time, only palliative care is available.

Life expectancy varies based on Sanfilippo type and individual progression, but is typically around 15 years.

Sanfilippo has its most devastating effects on the brain.

Children experience hyperactivity, sleeplessness, loss of speech, loss of toileting skills, intellectual disability, heart problems, vision loss, seizures, loss of mobility, dementia, and finally death.

The disease progression can vary significantly from one child to the next, making it particularly difficult to predict.

Symptoms cover a wide spectrum and some children may experience particular symptoms more than others.

Research to Cure Sanfilippo

Medical research has recently achieved promising break-throughs in gene therapy and enzyme-replacement therapy with hopes for the future. Clinical trials are happening or expected around the world. Information about clinical trials and referrals are available through the Foundation. For children living with Sanfilippo today, participation in a clinical trial is their only opportunity to try a treatment. Unfortunately, these trials only accept an extremely small number of patients, and time is not something these children have on their side. Cure Sanfilippo Foundation’s mission is to accelerate research to discovery a treatment or cure for Sanfilippo Syndrome.