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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 May 15, 2013 Ultragenyx Advances Clinical Development of UX003 for the Treatment of Mucopolysaccharidosis Type 7 (MPS 7)
Ultragenyx Advances Clinical Development of UX003 for the Treatment of Mucopolysaccharidosis Type 7 (MPS 7)
Toggle Summary Jul 3, 2013 Ultragenyx Announces a Positive Signal in Interim Data from Phase 2 Study of UX001 in Hereditary Inclusion Body Myopathy
Ultragenyx Announces a Positive Signal in Interim Data from Phase 2 Study of UX001 in Hereditary Inclusion Body Myopathy
Toggle Summary Sep 3, 2013 Ultragenyx Announces Collaboration with Kyowa Hakko Kirin to Develop and Commercialize Phase 2-stage KRN23 for X-linked Hypophosphatemia
Ultragenyx Announces Collaboration with Kyowa Hakko Kirin to Develop and Commercialize Phase 2-stage KRN23 for X-linked Hypophosphatemia
View all press releases »
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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.
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    • Message from BIO
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    • Crysvita®
    • Mepsevii™
    • Dojolvi™
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    • DTX 301 for OTC
    • DTX 401 for GSDIa
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    • XLH
    • TIO
    • MPS 7
    • LC-FAOD
    • OTC
    • GSD1a
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    • Access to Investigational Therapies
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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™
  • 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

      • Press Releases
      • Events & Presentations
      • Corporate Governance
      • SEC Filings
      • Stock Information
      • Analyst Coverage
      • Investor FAQ
      • Contact Us
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