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Alignment with FDA on Phase 3 study primary endpoint of Bayley-4 cognition and key secondary endpoint of Multi-Domain Responder Index (MDRI) Phase 3 study on track to initiate by the end of this...

NOVATO, Calif., June 20, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for...

NOVATO, Calif., June 13, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for...

NOVATO, Calif., June 12, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for...

Agreement reached with FDA that cerebral spinal fluid (CSF) heparan sulfate (HS) can be used as a reasonable surrogate endpoint for accelerated approval Data from existing clinical studies appear...

14-month data show treatment with setrusumab resulted in a large, sustained 67% reduction in annualized fracture rate and persistent median annualized fracture rate of 0.00 (p=0.0014) Treatment...

NOVATO, Calif., June 04, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for...

Treatment with DTX401 resulted in a statistically significant reduction in daily cornstarch intake at Week 48 (p<0.0001) with maintenance of glucose control Company will host investor call today...

Evkeeza is een eerste klasse geneesmiddel dat wordt aanbevolen voor vergoeding door het Nederlands Agentschap voor Gezondheidstechnologie voor de behandeling van HoFH bij patiënten ≥ 5 jaar oud ...

NOVATO, Calif., May 17, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for...