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Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

Press Releases

Date Title and Summary
Toggle Summary Feb 17, 2021 Ultragenyx to Present at Upcoming Investor Conferences
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 Emil D.
Toggle Summary Feb 17, 2021 Ultragenyx Announces Approval of Dojolvi™ (UX007/triheptanoin) in Canada for the Treatment of Long-chain Fatty Acid Oxidation Disorders in Adults and Children
First Approved Treatment for Adult and Pediatric Patients with Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) in Canada NOVATO, Calif., Feb. 17, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization
Toggle Summary Feb 11, 2021 Ultragenyx Reports Fourth Quarter and Full Year 2020 Financial Results and Corporate Update
2020 total revenue of $271.0 million and 2020 Crysvita 1 revenue to Ultragenyx of $138.9 million 2021 Crysvita 1 revenue in Ultragenyx territories guidance of $180 million to $190 million reaffirmed Strong Dojolvi launch continues with approximately 130 patients on reimbursed commercial therapy  in
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  • About
    • Management
      • Emil D. Kakkis, M.D., Ph.D.
      • Jayson Dallas, M.D.
      • Dennis Huang
      • Thomas Kassberg
      • Karah Parschauer
      • John Pinion
      • Shalini Sharp
    • Board of Directors
      • Daniel G. Welch
      • Emil D. Kakkis, M.D., Ph.D.
      • William Aliski
      • Deborah Dunsire, M.D.
      • Lars Ekman, M.D., Ph.D.
      • Matthew K. Fust
      • Michael Narachi
      • Clay B. Siegall, Ph.D.
    • Grants & Charitable Contributions
    • Our Commitment to Patients
    • Message from BIO
  • Medicines
    • Crysvita®
    • Mepsevii®
    • Dojolvi®
    • Dojolvi™ for Canada
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    • DTX 301 for OTC
    • DTX 401 for GSDIa
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    • XLH
    • TIO
    • MPS 7
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    • GSD1a
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  • About
    • Management
      • Emil D. Kakkis, M.D., Ph.D.
      • Jayson Dallas, M.D.
      • Dennis Huang
      • Thomas Kassberg
      • Karah Parschauer
      • John Pinion
      • Shalini Sharp
    • Board of Directors
      • Daniel G. Welch
      • Emil D. Kakkis, M.D., Ph.D.
      • William Aliski
      • Deborah Dunsire, M.D.
      • Lars Ekman, M.D., Ph.D.
      • Matthew K. Fust
      • Michael Narachi
      • Clay B. Siegall, Ph.D.
    • Grants & Charitable Contributions
    • Our Commitment to Patients
    • Message from BIO
    • Medicines
      • Crysvita®
      • Mepsevii®
      • Dojolvi®
      • Dojolvi™ for Canada
  • Pipeline
    • DTX 301 for OTC
    • DTX 401 for GSDIa
    • Presentations & Publications
  • For Patients
    • XLH
    • TIO
    • MPS 7
    • LC-FAOD
    • OTC
    • GSD1a
    • Access to Approved Therapies
    • Access to Investigational Therapies
  • Investors

      Investors

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