What is the Prognosis of Sanfilippo Syndrome?

Sanfilippo is devastating to the brain and body.

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.

Life expectancy of children with Sanfilippo

At this time, only palliative care is available. Life expectancy varies based on Sanfilippo type and individual progression. The average life expectancy is in the teens.A 2017 study of patients in the United Kingdom with Sanfilippo syndrome analyized lifespans according to type of Sanfilippo and found:

  • In Sanfilippo type A: mean age at death (± standard deviation) was 15.22 ± 4.22 years,
  • In Sanfilippo type B: mean age at death was 18.91 ± 7.33 years
  • In Sanfilippo type C: mean age at death was 23.43 ± 9.47 years
  • Sanfilippo type D was not analyzed because the study lacked data on patients with this rare type

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. Some children may never experience certain symptoms.

One family’s experience with Sanfilippo Syndrome

This is the Jessop family’s story, sharing their oldest son Dylan’s journey with Sanfilippo Syndrome, particularly highlighting how the disease progressed over time.

Sanfilippo Does Not Have a Cure … Yet.

At this time, only supportive or “palliative” care is available. Life expectancy varies based on Sanfilippo type and individual progression, but is typically around 15 years. 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. Medical research has recently achieved promising break-throughs with real hopes for the future, but we are not there yet.

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