How Sanfilippo Syndrome Type B Affects Children
Some call it a childhood version of Alzheimer’s disease. Sanfilippo syndrome type B is a rare neurodegenerative disease that, due to a missing enzyme, compromises the body’s ability to break down heparan sulfate. The result is a build-up of toxins in the central nervous system. With an estimated incidence of 1:150,000, the disease affects at least 4,200 children worldwide and no approved treatments exist.
When the disease begins, affected children may have difficulty acquiring language or experience frequent ear or sinus infections.
As affected children age, cognitive impairment, behavioral symptoms and sleep difficulties develop. Eventually, patients experience cognitive decline, difficulty walking, difficulty eating and seizures.
These symptoms are associated with many childhood diseases, which delays diagnosis of Sanfilippo syndrome type B.
By developing an effective treatment that can slow or stabilize clinical manifestations—and raising awareness needed for earlier diagnosis—we believe these children can have improved health and quality of life.
Discover the resources available to support children with Sanfilippo syndrome type B and their caregivers. If you want to learn about testing, diagnosis, and symptoms of Sanfilippo syndrome type B, talk with your child’s doctor.