The U.S. Food and Drug Administration (FDA) is once again considering whether or not it will approve UX111, Ultragenyx Pharmaceutical’s experimental gene therapy for Sanfilippo syndrome type A. According to Ultragenyx, the FDA has accepted a resubmitted biologics license application (BLA), and a decision from the agency is expected…
Discussion
As a parent, I’ve met other moms who are proud that their children are “screen-free,” meaning they aren’t allowed to watch television on a tablet. I can’t relate in the least. My 6-year-old daughter, Liv, thrives with screen time. She has a degenerative genetic disorder called Sanfilippo syndrome, which…
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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