Olivia had been showing symptoms since she was just 7 months old — including a belly that remained swollen, hard to the touch ...

The FDA cited manufacturing issues but did not flag problems with Ultragenyx’s data package for UX111, with the biotech ...

Ultragenyx Pharmaceuticals RARE announced that the FDA has issued a Complete Response Letter (CRL) for its biologics license ...

Sanfilippo syndrome is a rare genetic disorder that affects the body's ability to break down complex sugars, causing symptoms of dementia in children. There is no cure, and the regression is ...

The effects of Sanfilippo syndrome in small children are so devastating that the rare disease is often known as "childhood dementia." About one in 70,000 newborns inherit this disorder.

Ultragenyx Pharmaceuticals took another hit when the FDA rejected its gene therapy for patients with a rare, genetic disorder.

The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat ...

In December 2021, Newsweek shared the story of Simon Croke – a 5-year-old boy diagnosed with Sanfilippo Syndrome, a fatal degenerative disease that presents symptoms similar to Alzheimer's. On ...

This is because on March 28, Liv was diagnosed with Sanfilippo Syndrome Type B, a rare disease that is compared to late-stage Alzheimer's, but for children. "It is 100% fatal," said Erin.