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

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

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

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

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

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

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

Novato, California Monday, July 14, 2025, 16:00 Hrs [IST] ...

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

Manufacturing issues are the latest problem for Ultragenyx Pharmaceutical Inc. to solve after last week’s disappointment in a phase III study to treat brittle bones. The U.S. FDA gave the company a ...

The Sanfilippo Children’s Foundation notes: “As the brain gets progressively damaged, children experience severe hyperactivity, disordered sleep, loss of speech, cognitive decline, cardiac issues, ...

The firm said the agency cited chemistry, manufacturing, and controls issues in its complete response letter, but had no concerns about product quality.