As morbid as it sounds, I sometimes find myself thinking about my daughter’s death and funeral. In 2022, Liv was diagnosed with Sanfilippo syndrome, a terminal, neurodegenerative, genetic disorder. Today, she is 6 and doing well, all things considered, but this diagnosis has felt like nothing short of a…
Discussion
Long-term treatment with the investigational enzyme replacement therapy tralesinidase alfa stabilized cognitive function for up to six years in children with Sanfilippo syndrome type B, clinical trial data showed. Spruce Biosciences, the therapy’s developer, plans to use the data to support an application seeking accelerated approval of the…
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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