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NOVATO, Calif., April 22, 2021 (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 announced the successful completion of an End-of-Phase 2 ...
Gene therapy concern Solid Biosciences looks to be taking a meaningful step forward in its Phase 1/2 Duchenne muscular dystrophy trial, after dosing only seven patients in 3 1/2 years. Although Sarepta Therapeutics (SRPT) suffered a setback with its therapy in January 2021, Pfizer...
Foundation’s collaboration with premier rare disease company strengthens efforts to reach more patients in need n-Lorem Foundation , a nonprofit organization that provides free, lifetime supplies of individualized RNA-targeted medicines to patients living with ult...
Ultragenyx Pharmaceutical (RARE) announces that the U.S. FDA has given it the go-ahead to proceed with its Investigational New Drug ((IND)) application for UX053, an investigational mRNA therapy being evaluated for the treatment of Glycogen Storage Disease Type III (GSDIII).Enrollment in a Ph...
NOVATO, Calif., March 08, 2021 (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 the U.S. Food and Drug Admi...
MREO came to some prominence after Ultragenyx gave it a nice deal in December 2020. Its lead asset etigilimab has some early interesting data. It will take a few more years for the company to garner more investor interest. For further details see: Mereo BioPharma: Underf...
Ultragenyx Pharmaceutical (RARE) announces that Health Canada has approved Dojolvi (triheptanoin) as a source of calories and fatty acids for the treatment of adult and pediatric patients with long-chain fatty acid oxidation disorders (LC-FAOD).Long-chain fatty acid oxidation disorders (LC-FA...
NOVATO, Calif., Feb. 17, 2021 (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 Health Canada has approved D...
In Q4 2020 results, Ultragenyx Pharmaceutical (RARE) beat the street forecast in terms of both revenue and EPS.However, noting the outperformance of the company shares in recent months, JPMorgan has downgraded the stock to neutral from overweight.The analyst Cory Kasimov has raised the p...
The following slide deck was published by Ultragenyx Pharmaceutical Inc. in conjunction with their 2020 Q4 earnings call. For further details see: Ultragenyx Pharmaceutical Inc. 2020 Q4 - Results - Earnings Call Presentation
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NOVATO, Calif., July 25, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, today announced that it will host a conference call at 5...
NOVATO, Calif., July 24, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the company will share the latest clinical data, regulatory progress and program next steps for GTX-102, its investigational antisense oligonucleotide for Angelman syndrome, on Wed...
2024-07-24 04:06:00 ET Stock Traders Daily has produced this trading report using a proprietary method. This methodology seeks to optimize the entry and exit levels to maximize results and limit risk, and it is also applied to Index options, ETFs, and futures for our subscribers. This...