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Expanded Label Includes Additional Clinical Data Highlighting Superiority Over Conventional Therapy for Pediatric Patients and Extends Current U.S. Indication to Patients Aged Six Months and Older Expanded Label Includes Data Demonstrating Improvement in Stiffness, Continued Further H...
Introduction Ultragenyx Pharmaceuticals ( RARE ) is a mid-cap ($2.46B) biopharma creating and developing innovative therapeutics for ultra-rare and/or orphan as well as genetic diseases. CEO Dr. Emil Kakkis has had a lifelong research career in developing novel therapeutics for rare diseas...
Ultragenyx (NASDAQ: RARE ) and collaboration partner Kyowa Kirin ( OTCPK:KYKOF ) announce plans to submit a supplemental marketing application to the FDA in H1 2020 seeking approval to use Crysvita (burosumab) to treat FGF23-related hypophosphatemia associated with phosphaturic mesenc...
NOVATO, Calif. and TOKYO, Sept. 10, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, and Kyowa Kirin Co., Ltd., (Kyowa Kirin, TYO: 4151) today announced plans to...
Ultragenyx Pharmaceutical (NASDAQ: RARE ) is up 10% premarket on light volume in reaction to positive data from the second cohort in its Phase 1/2 clinical trial evaluating gene therapy DTX401 in patients with glycogen storage disease type Ia. The data were presented at SSIEM...
Increased Time to Hypoglycemia and Reduction in Cornstarch Use in Cohorts 1 and 2; Improvement of Additional Key Metabolic Measures Observed Cohort 1 Patients Continue to Demonstrate Long-Term, Durable Responses Company to Enroll Three Patients in Expansion Cohort to Confirm 6.0 ...
SARASOTA, Fla. and NOVATO, Calif., Sept. 03, 2019 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, today announced that the U.S...
Ultragenyx Pharmaceuticals (NASDAQ: RARE ) will partner with privately held GeneTx Biotherapeutics LLC to develop the latter's GTX-102, an antisense oligonucleotide, for the treatment of rare inherited nervous system disorder called Angelman syndrome . More news on: Ultragenyx Pharmac...
Program aims to be first disease-modifying treatment for this serious neurogenetic disorder Ultragenyx receives an exclusive option to acquire GeneTx NOVATO, Calif. and DOWNERS GROVE, Ill., Aug. 14, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmac...
NOVATO, Calif., Aug. 13, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Tom Kassberg, the company’s Chief Business ...
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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 ...