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 ...

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

While the CRL derails a near-term clearance, one analyst described the rejection as more of “a speed bump to approval, rather ...

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.

The FDA's request stems from observations made during recent inspections of Ultragenyx's manufacturing facilities.

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 ...

Around 3,500 FDA employees received termination emails; FDA Commissioner Marty Makary suggests lowering industry user fees ...

Ultragenyx received a Complete Response Letter from the FDA for UX111 AAV gene therapy to treat Sanfilippo Syndrome Type A ...