RARE News

NIO, Ultragenyx Pharmaceutical and McDonald's slipped to annual lows on Thursday amid company-related pressures and Wall Street pessimism around the stocks.

Zanvastro is now approved for children and adults with Alexander disease, a rare and often fatal brain condition that affects fewer than one in a million people.

Ultragenyx (RARE) reported earnings 30 days ago. What's next for the stock? We take a look at earnings estimates for some clues.

Ultragenyx plunges 46% after its phase III Angelman syndrome study fails to achieve key efficacy goals, putting apazunersen's future in question.

Futures Flat As Yields Ignore Oil Meltup Futures are flats with Tech in line and small caps lagging even as bond yields dip 1bp across the curve, ignoring the continued rise in oil. In a quiet session ahead of tomorrow's jobs report,?Japan remains the standout, with the yen...

William Blair downgraded Ultragenyx to ‘Market Perform’ from ‘Outperform,’ stating the miss could have negative implications for Ultragenyx’s other Angelman syndrome trial.

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.