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Tralesinidase alfa, an experimental enzyme replacement therapy for people with Sanfilippo syndrome type B, was safe and showed good brain distribution in non-human primates, specifically cynomolgus monkeys, a new study found. The treatment was equally well tolerated regardless of dose, duration of the infusion, or formulation. According to…
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To many of you, it’s May. To those of you with school-aged children, it’s “Maycember” — that hectic time at the end of the school year. To me, it’s also less than a month until my baby boy, Will, turns 13. We are fully immersed in Maycember here.
With a sister who has Sanfilippo, columnist Emily Wallis cherishes her memories, holds the present tight, and awaits the next chapter.
Check out Sanfilippo Syndrome News’ collection of health insights, where you can find a variety of information relevant for patients and caregivers.
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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