Ultragenyx said the late-stage Aspire study of apazunersen, also known as GTX-102, did not meet its primary goal or key backup goal in a late-stage trial.

ALNY, BMRN, RARE and BBIO offer rare-disease growth potential through strong products, pipeline catalysts and high unmet medical needs.

Ultragenyx stock rises as Genglycos wins FDA's accelerated approval as the first treatment targeting the underlying cause of GSDIa, boosting its rare-disease portfolio.

Analysts raised their price targets on RARE stock after the FDA granted accelerated regulatory approval for its rare metabolic disease therapy.

Futures Slide As Treasury Yields Surge, Erasing Bessent Intervention, Driven By Oil Spike US futures slide and are trading at session lows,?as bond yields surge after yesterday's Treasury announcement, having now erased the entire post buyback-boost move; yields are 4-5bps higher as...

The FDA granted accelerated approval to GENGLYCOS, also called DTX401, for patients aged eight and older with glycogen storage disease type Ia (GSDIa).

The FDA is due to rule by August 23 on Ultragenyx’s application for DTX401, a one-time gene therapy for von Gierke disease.

RARE beats Q2 earnings estimates as revenues surge year over year to a record quarterly high, driven by higher product sales across its portfolio.

While the top- and bottom-line numbers for Ultragenyx (RARE) give a sense of how the business performed in the quarter ended June 2026, it could be worth looking at how some of its key metrics compare to Wall Street estimates and year-ago values.