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Clinical-stage monoclonal antibody in development for rare genetic bone disease that builds on Ultragenyx’s existing bone franchise Ultragenyx leads and funds development to approval; Mereo retains commercial rights in Europe, Ultragenyx commercializes in US and in rest o...
Commenting on the proposed merger between Alexion (ALXN) and AstraZeneca (AZN), Piper Sandler highlights the ‘durability’ of large biotech businesses, ‘with Astra pointing to double-digit EPS accretion in years 1-3’ from the deal.“If the market doe...
The FDA designates Orphan Drug status to Ultragenyx Pharmaceutical's (RARE) UX701 for the treatment of Wilson disease, a rare metabolic disease in which the body is unable to transport copper, thus accumulation of copper in the liver and other tissues. It is caused by mutations in the AT...
NOVATO, Calif., Dec. 09, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare genetic diseases, today announced that the U.S. Food and Drug Administratio...
Ultragenyx Pharmaceutical Inc. (RARE), announcing the data from the Phase 1/2 study of GTX-102 in Angelman syndrome, says the additional data, including EEG findings, support prior initial indications of activity with no new adverse events.In August 2019, Ultragenyx partnered with G...
Results presented at Foundation for Angelman Syndrome Therapeutics (FAST) Global Summit Additional data , including EEG findings, support prior initial indications of activity and there were no new adverse events SARASOTA, Fla. and NOVATO, Calif., Dec. ...
Sigilon Therapeutics has filed proposed terms for its $101 million U.S. IPO. The firm is advancing programs to treat blood, lysosomal storage and other diseases. SGTX has produced a positive initial result with its first trial patient, has a strong collaboration relationship with ...
NOVATO, Calif., Nov. 24, 2020 (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 Emil D. Kakkis, M.D., Ph.D.,...
Sigilon Therapeutics has filed to raise capital via an IPO. The firm is developing treatments for blood disorders and other diseases. SGTX has produced intriguing preclinical study results for its lead candidate for the treatment of Hemophilia A. For further details see: ...
As the demand for a COVID-19 vaccine or a treatment is on the rise with the number of coronavirus cases increasing each passing day, Alexion Pharmaceuticals (ALXN), BioNTech (BNTX), and Ultragenyx Pharmaceutical (RARE) should benefit significantly because of the progress they made so far with...
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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...