Physical therapy (PT) appears to be safe for children with Sanfilippo syndrome and other types of mucopolysaccharidosis (MPS), and may help maintain function, ease pain, and support quality of life, according to a small study. However, the researchers said, many families were unable to attend all scheduled therapy sessions, underscoring…
Discussion
“What would Liv want?” This is a question I ask myself every day. Our 6-year-old daughter, Liv, has a neurodegenerative genetic disorder called Sanfilippo syndrome. She can no longer say yes or no, point to what she wants, or even nod her head. What does she want to wear?…
Sanfilippo syndrome is a genetic disorder that leads to a form of childhood dementia. Learn more about the disease here.
Sanfilippo is caused by mutations in one of four genes that produce the enzymes needed to break down heparan sulfate. Learn more about what causes the disease here.
Symptoms range from behavioral problems to developmental delays and sleep issues. Learn more about the symptoms of the disease here.
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