公司简介
Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment...
关联新闻
- US FDA approves Ultragenyx's gene therapy for rare disorder
The U.S Food and Drug Administration said on Thursday it has approved Ultragenyx's gene therapy for the treatment of a rare, fatal disease.
- Ultragenyx's rare disease drug fails in late-stage trial; shares plunge
Ultragenyx Pharmaceutical said on Wednesday its experimental drug for a rare genetic neurodevelopmental disorder did not meet the main goal in a late-stage trial, sending its shares down 40% in af...
- Ultragenyx's gene therapy for rare metabolic disorder secures US FDA nod
The U.S. Food and Drug Administration approved Ultragenyx Pharmaceutical's gene therapy to treat a rare metabolic disorder, the company said on Wednesday.