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4 Rare Disease Drugmakers With Strong Long-Term Growth Prospects
ALNY, BMRN, RARE and BBIO offer rare-disease growth potential through strong products, pipeline catalysts and high unmet medical needs.
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RARE Stock Gains on FDA Nod for Rare Disease Gene Therapy Genglycos
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.
Zacks
Ultragenyx’s Gene-Therapy Win Could Unlock A ‘Long Revenue Tailwind,’ Says Wells Fargo — This Analyst Sees 265% Upside For RARE Stock
Analysts raised their price targets on RARE stock after the FDA granted accelerated regulatory approval for its rare metabolic disease therapy.
Stocktwits
Futures Slide As Treasury Yields Surge, Erasing Bessent Intervention, Driven By Oil Spike
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...
Zero Hedge
RARE Stock Jumps 6% After-Hours On First Gene Therapy Approval, New Priority Review Voucher
The FDA granted accelerated approval to GENGLYCOS, also called DTX401, for patients aged eight and older with glycogen storage disease type Ia (GSDIa).
Stocktwits
RARE Stock Clocks Two Consecutive Days Of Losses Ahead Of Weekend FDA Decision On Gene Therapy
The FDA is due to rule by August 23 on Ultragenyx’s application for DTX401, a one-time gene therapy for von Gierke disease.
Stocktwits
Ultragenyx's Q2 Earnings Beat Estimates, Revenues Jump Y/Y
RARE beats Q2 earnings estimates as revenues surge year over year to a record quarterly high, driven by higher product sales across its portfolio.
Zacks
Ultragenyx (RARE) Q2 Earnings: Taking a Look at Key Metrics Versus Estimates
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.
Zacks
Ultragenyx (RARE) Reports Q2 Loss, Tops Revenue Estimates
Ultragenyx (RARE) delivered earnings and revenue surprises of +29.13% and +18.23%, respectively, for the quarter ended June 2026. Do the numbers hold clues to what lies ahead for the stock?
Zacks
Ultragenyx Q2 Loss Narrows, Reaffirms 2026 Revenue Outlook
(RTTNews) - Ultragenyx Pharmaceutical Inc. (RARE) on Tuesday reported a narrower net loss for the second quarter, as revenues grew from a year ago. The company reaffirmed its full-year 2026 revenue guidance...
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