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

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.

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

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

Sanfilippo syndrome is a terminal disease with no cure and no treatment. It affects one in 70,000 children and is a genetic condition that affects the metabolism of complex molecules and results ...

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.

After 4-year-old Eliza O'Neill was diagnosed last summer with Sanfilippo syndrome, a rare and fatal genetic disease, her parents, Glen and Cara, realized the real heartbreak wasn't actually the ...

HOUSTON — Sanfilippo Syndrome — also known as Mucopolysaccharidosis type III or MPS III — is a terminal, neurodegenerative rare disease. It causes children to lose all the skills they've ...

Eliza has Sanfilippo syndrome type A, a rare genetic disorder that causes a deadly buildup of heparin sulfate in her cells. Soon, Eliza will lose the ability to speak, her parents say.

Eliza suffers from Sanfilippo syndrome–Type A, a rare and ultimately fatal genetic disorder that primarily affects the brain. The 5-year-old is slowly losing her ability to speak, ...