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If approved, UX111 will be the first approved therapy for the treatment of Sanfilippo syndrome Type A, a rare disease affecting young children that leads to progressive, irreversible neurodegeneration...
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NOVATO, Calif., March 30, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New...
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NOVATO, Calif., March 20, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for...
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Statistically significant improvements in primary endpoint of ammonia control compared with placebo at 36 weeks Clinically important changes observed in patient global impression scale for overall...
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NOVATO, Calif., Feb. 23, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for...
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PDUFA action date set for August 23, 2026 If approved, DTX401 will be the first treatment to address the underlying cause of GDSIa NOVATO, Calif., Feb. 23, 2026 (GLOBE NEWSWIRE) -- Ultragenyx...
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NOVATO, Calif., Feb. 20, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for...
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2025 total revenue of $673 million,Crysvita® revenue of $481 million and Dojolvi® revenue of $96 million 2026 total revenue from current products expected to be between $730 million to $760 million...
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NOVATO, Calif., Feb. 05, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for...
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Data represents up to 8.5 years of follow-up and are consistent across age, dose, and genotype BLA resubmitted to U.S. FDA in January 2026; Company expects up to six-month review period per FDA...