Group 1 - Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is recognized as a promising biotech stock, with Assenagon Asset Management increasing its holdings by 439.0% in Q2, acquiring 947,865 shares for a total investment of $42,314,000, representing about 1.23% ownership [1] - The company has received Breakthrough Therapy designation for GTX-102 in Angelman syndrome and is positioned to advance UX143, which is expected to be a transformative treatment for osteogenesis imperfecta in both pediatric and adult patients [2] - Strategic collaborations, including a recent FDA approval for Regeneron's Evkeeza, indicate a positive outlook for Ultragenyx, despite a recent 26% drop in stock price due to a failed treatment approval [3] Group 2 - Ultragenyx is a California-based biopharmaceutical company focused on discovering, developing, and commercializing treatments for rare and ultra-rare genetic diseases, with key products including Crysvita, Mepsevii, Dojolvi, and Evkeeza [4]

First and only medicine approved by Health Canada for patients as young as 6-months of age with HoFH, an ultrarare, inherited form of high cholesterol TORONTO, July 14, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialisation of novel therapies for rare and ultra-rare genetic diseases, today announced that Health Canada has extended the approval of Evkeeza (evinacumab) as an adjunct to diet and other low-density lipo ...