Remove my roles of pharmacist, wife, and a mom to two kids, and who am I? What else defines me? This was the question presented to the parents of kids with Sanfilippo syndrome in a group therapy session recently. All the parents in the group have children with the…
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…
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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