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Ultragenyx is a clinical-stage biopharmaceutical company committed to bringing to market novel products for the treatment of rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. Founded in 2010, the company has rapidly built a diverse portfolio of product candidates with the potential to address diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are no approved therapies.

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 Jun 20, 2011 Ultragenyx Announces Series A Financing
Ultragenyx Announces Series A Financing
Toggle Summary Aug 15, 2011 Ultragenyx Initiates Phase 1 Clinical Study of UX001 in Hereditary Inclusion Body Myopathy (HIBM), a Rare Muscle Wasting Disease
Ultragenyx Initiates Phase 1 Clinical Study of UX001 in Hereditary Inclusion Body Myopathy (HIBM), a Rare Muscle Wasting Disease
Toggle Summary Oct 5, 2011 Ultragenyx Granted Orphan Drug Designation for UX001 SA-ER for the Treatment of Hereditary Inclusion Body Myopathy (HIBM)
Ultragenyx Granted Orphan Drug Designation for UX001 SA-ER for the Treatment of Hereditary Inclusion Body Myopathy (HIBM)
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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
    • Message from BIO
  • Medicines
    • Crysvita®
    • Mepsevii™
  • Pipeline
    • Burosumab for XLH
    • Burosumab for TIO
    • Vestronidase alfa
    • UX007 for FAOD
    • UX007 for Glut1 DS
    • DTX 301 for OTC
    • DTX 401 for GSD1a
    • Presentations & Publications
  • For Patients
    • XLH
    • TIO
    • MPS 7
    • LC-FAOD
    • Glut1 DS
    • OTC
    • GSD1a
    • 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
    • Message from BIO
    • Medicines
      • Crysvita®
      • Mepsevii™
  • Pipeline
    • Burosumab for XLH
    • Burosumab for TIO
    • Vestronidase alfa
    • UX007 for FAOD
    • UX007 for Glut1 DS
    • DTX 301 for OTC
    • DTX 401 for GSD1a
    • Presentations & Publications
  • For Patients
    • XLH
    • TIO
    • MPS 7
    • LC-FAOD
    • Glut1 DS
    • OTC
    • GSD1a
    • Access to Approved Therapies
    • Access to Investigational Therapies
  • Investors

      Investors

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