Ultragenyx Pharmaceutical Inc.
NEWS
Ultragenyx Pharmaceutical Inc. and Kyowa Kirin Co., Ltd. announced plans to submit a supplemental Biologics License Application to the U.S. Food and Drug Administration for Crysvita® for the treatment of FGF23-related hypophosphatemia associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized.
Ultragenyx Pharmaceutical Inc. announced positive data from the second dose cohort of the ongoing Phase 1/2 study of DTX401, an adeno-associated virus based gene therapy for the treatment of glycogen storage disease type Ia.
GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation and Rare Pediatric Disease Designation to GTX-102 for the treatment of Angelman Syndrome, a serious, debilitating rare neurogenetic disorder that affects approximately 1 in 15,000 people worldwide.
Ultragenyx Pharmaceutical Inc. announced that Shalini Sharp, the company’s Chief Financial Officer, will present at the following conferences
Under the terms of the deal, Utragenyx will pay GeneTx $20 million for an exclusive option to acquire GeneTx.
Ultragenyx Pharmaceutical Inc. and GeneTx Biotherapeutics LLC announced a partnership to develop GeneTx’s GTX-102, an antisense oligonucleotide (ASO) for the treatment of Angelman syndrome, a serious, debilitating, rare neurogenetic disorder that affects approximately 1 in 15,000 people worldwide.
Ultragenyx Pharmaceutical Inc. announced that Tom Kassberg, the company’s Chief Business Officer, will present at the Wedbush PacGrow Healthcare Conference on Wednesday, August 14, 2019 at 1:20 PM ET in New York, NY.
Ultragenyx Pharmaceutical Inc. reported its financial results and corporate update for the quarter ended June 30, 2019.
Ultragenyx Pharmaceutical Inc. announced that it has submitted to the U.S. Food and Drug Administration a New Drug Application for UX007 for the treatment of long-chain fatty acid oxidation disorders, a group of genetic disorders in which the body is unable to convert long-chain fatty acids into energy.
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