Ultragenyx Pharmaceutical Inc (RARE) USD0.001
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Business summary
Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company. The Company is focused on the identification, acquisition, development, and commercialization of novel products for the treatment of serious rare and ultrarare genetic diseases. Its therapies and clinical-stage pipeline consist of four product categories: biologics, small molecules, AAV gene therapy, and nucleic acid product candidates. The Company’s four approved product candidates include Crysvita (burosumab) for the treatment of X-linked hypophosphatemia (XLH), and tumor-induced osteomalacia (TIO), Mepsevii (vestronidase alfa) for the treatment of mucopolysaccharidosis VII (MPSVII) or Sly Syndrome, Dojolvi (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD), and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia (HoFH). Its clinical product candidates include DTX401, DTX301, and UX701. DTX401 for the treatment of glycogen storage disease type la.
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General stock information
- Short code:
- RARE
- ISIN:
- US90400D1081
- Market cap:
- $4.04 billion
- Shares in issue:
- 92.34 million
- Sector:
- Biotechnology
- Exchange:
- NASDAQ
- Country:
- United States
- Currency:
- US dollar
- Indices:
- NASDAQ Comb. Composite
Key personnel
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