A single dose of UX111 (rebisufligene etisparvovec), an experimental gene therapy being developed by Ultragenyx Pharmaceutical, reduces the toxic buildup of heparan sulfate for more than eight years and helps children with Sanfilippo syndrome type A maintain or improve developmental skills, particularly when administered early. These long-term results…
Discussion
Three years ago, a random dog roamed into our backyard, and a minute later, a woman arrived chasing after him. My husband helped corral the dog, the owner thanked him, and we went on with our day. A few days later, the woman reached out online after seeing the “Saving…
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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