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NOVATO, Calif., Feb. 06, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced data demonstrating treatment with UX111 (ABO-102) AAV gene therapy resulted in rapid and sustained decreased levels of heparan sulfate (HS) in cerebrospinal fluid (CSF) in patients with...
GTX-102 is the first Angelman syndrome therapeutic candidate to receive PRIME designation Phase 1/2 study fully enrolled; expansion data expected in first half of 2024 NOVATO, Calif., Feb. 05, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced t...
Safety and initial efficacy data from Stage 1 expected in the first half of 2024 Dose selection and initiation of Stage 2 to follow in the second half of 2024 NOVATO, Calif., Jan. 25, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that all p...
2024-01-19 09:45:01 ET Wells Fargo analyst issues OVERWEIGHT recommendation for RARE on January 19, 2024 10:00AM ET. The previous analyst recommendation was Overweight. RARE was trading at $43.77 at issue of the analyst recommendation. The overall analyst consensus : BUY...
Phase 3 portion of Orbit Study and Phase 3 Cosmic Study of setrusumab for treatment of Osteogenesis Imperfecta (OI) conducted by partner Ultragenyx expected to complete enrollment around the end of 1Q 2024 and 1H 2024, respectively; Additional Phase 2 data expected in 2024 Alignment on de...
Preliminary 2023 Total Revenue of $430 million to $435 million, Crysvita® Revenue of $325 million to $330 million and Dojolvi® revenue of $70 million to $71 million 2024 expected Total Revenue guidance between $500 million to $530 million, Crysvita revenue of $375 millio...
NOVATO, Calif., Jan. 04, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the National Institute for Health and Care Excellence (NICE) has issued a final draft guidance recommending Evkeeza® (evinacumab) to NHS England. Evkeeza is recommended within...
NOVATO, Calif., Jan. 03, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today announced the completion of patient enrollment in...
NOVATO, Calif., Jan. 02, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the c...
2023-12-24 09:48:14 ET Summary Ultragenyx Pharmaceuticals' shares are trading 60% higher after pricing a secondary offering, reflecting optimism about its pipeline of rare disease treatments. The company has four commercial products, with Crysvita generating the majority of its re...
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NOVATO, Calif., May 07, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the develo...
NOVATO, Calif., May 07, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today announced that Eric Crombez, M.D., the company's c...
First quarter total revenue of $109 million, Crysvita® revenue of $83 million and Dojolvi® revenue of $16 million Reaffirmed 2024 expected total revenue guidance between $500 million to $530 million, Crysvita revenue of $375 million to $400 million, and Dojolvi revenue of $75 ...