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…
Discussion
I am now at peace with my daughter Liv’s terminal, neurodegenerative, genetic disorder. Over four years ago, at age 2, she was diagnosed with Sanfilippo syndrome. Liv was still gaining skills at the time, but we were told that soon, it would shift, and she would start regressing and…
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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