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 ...

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

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the best oversold NASDAQ stocks to buy now. On July 14, William Blair ...

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.

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 ...

Torrance, California, June 21, 2024 (GLOBE NEWSWIRE) -- As a neurodegenerative disease characterized by childhood onset dementia, Sanfilippo syndrome causes immense suffering in many ways ...

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, ...