FAYUVI is a highly anticipated, first-ever treatment option with the potential to stop or slow the devastating, irreversible neurologic progression and loss of function associated with Sanfilippo syndrome Type A Ultragenyx’s UltraCare® program will support access, and commercial product is expected
Phase 3 Aspire did not achieve the primary endpoint of change from Baseline in Bayley-4 cognitive raw score nor the key secondary endpoint of net response in Multidomain Responder Index (MDRI) NOVATO, Calif., Sept. 02, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc.
At Week 96, participants across treatment and crossover groups experienced mean reduction in daily cornstarch intake of 61% while maintaining glycemic control, with most participants achieving reduction of at least 50% Complete elimination of nighttime cornstarch dosing observed in 33% of DTX401
NOVATO, Calif., Aug. 25, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of 45,984 restricted stock units of the
GENGLYCOS is the first gene therapy approval, and fifth FDA approval overall, for the company Approval provides a long-awaited first-ever option to reduce the burden of care associated with GSDIa Ultragenyx received a Priority Review Voucher upon approval Ultragenyx to host conference call on
Second quarter total revenue of $214 million, Crysvita ® revenue of $156 million and Dojolvi ® revenue of $27 million Reaffirm 2026 financial guidance, including total revenue of $730 million to $760 million and combined R&D and SG&A expenses to be flat to slightly down versus 2025; remain on path
NOVATO, Calif., July 28, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call at
NOVATO, Calif., July 24, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of 65,886 restricted stock units of the
NOVATO, Calif., July 08, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) released its 2025 Impact Report, detailing the company's work on behalf of patients and families living with rare and ultra-rare diseases. In 2025, Ultragenyx advanced five investigational therapies in