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

Complete Response Letter (CRL) cited specific chemistry, manufacturing and controls (CMC) related observations that are ...

The MPS panel tested for several rare diseases, including Hunter syndrome, Hurler syndrome, Morquio syndrome — and Sanfilippo syndrome, a particularly devastating disorder known for neurological ...

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

As Erin explains, Sanfilippo syndrome affects every aspect of Liv’s life. Liv, who's now 4 years old, has sleep issues — getting her to fall asleep, stay asleep and remain contained in her room ...

Ultragenyx Pharmaceutical (Nasdaq: RARE) has been dealt a regulatory setback, as the American medicines regulator has ...

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the 11 Best All-Time Low Stocks to Buy According to Analysts. On July ...

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

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

Second-quarter earnings come amid many high-level challenges for the biopharma industry. How will these five closely watched ...

Denali's lead drug for Hunter syndrome wins FDA Priority Review, marking a key step toward commercial-stage status.

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 ...