Ultragenyx (RARE) Reports Q1 Earnings: What Key Metrics Have to Say
https://www.zacks.com/stock/news/2267565/ultragenyx-rare-reports-q1-earnings-what-key-metrics-have-to-say?cid=CS-ZC-FT-fundamental_analysis|nfm-2267565
May 02, 2024 - While the top- and bottom-line numbers for Ultragenyx (RARE) give a sense of how the business performed in the quarter ended March 2024, it could be worth looking at how some of its key metrics compare to Wall Street estimates and year-ago values.
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Harmony Biosciences acquires Epygenix gaining rare epilepsy pipeline
https://seekingalpha.com/news/4096206-harmony-biosciences-acquires-epygenix-gaining-rare-epilepsy-pipeline?source=feed_sector_healthcare
Apr 30, 2024 - Harmony Biosciences acquires Epygenix Therapeutics for $35M upfront, focusing on developing treatments for rare epilepsy conditions. Read more here.
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FDA approves Pfizer’s first gene therapy for rare inherited bleeding disorder
https://www.cnbc.com/2024/04/26/fda-approves-pfizer-gene-therapy-beqvez-for-treatment-of-hemophilia-b.html
Apr 26, 2024 - The agency greenlighted the drug, which will be marketed as Beqvez, for adults with moderate to severe hemophilia B.
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