Rare genetic disease treatment

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Rafael Holdings Reports Third Quarter Fiscal 2025 Financial Results
GlobeNewswire News Room· 2025-06-11 11:00
NEWARK, N.J., June 11, 2025 (GLOBE NEWSWIRE) -- Rafael Holdings, Inc. (NYSE: RFL; NYSE American: RFL-WT), today reported its financial results for the third quarter and first nine months of fiscal year 2025 ended April 30, 2025. “We are pleased to have completed our merger with Cyclo Therapeutics and look forward to reporting the topline data from the 48-week interim analysis of the pivotal Phase 3 TransportNPC™ study evaluating Trappsol® Cyclo™ for the treatment of Niemann-Pick Disease Type C1 anticipated ...
Ultragenyx to Participate at Goldman Sachs 46th Annual Global Healthcare Conference
Globenewswire· 2025-06-03 21:00
Company Overview - Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company focused on developing and commercializing novel therapies for serious rare and ultra-rare genetic diseases [3] - The company has established a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical needs [3] Management and Strategy - The management team of Ultragenyx is experienced in the development and commercialization of therapeutics for rare diseases [4] - The company's strategy emphasizes time- and cost-efficient drug development to deliver safe and effective therapies to patients urgently [4] Upcoming Events - Emil Kakkis, M.D., Ph.D., the CEO of Ultragenyx, will participate in a fireside chat at the Goldman Sachs 46th Annual Global Healthcare Conference on June 10, 2025, at 10:00 a.m. ET [1] - A live and archived webcast of the panel will be available on the company's website [2]