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Transforming good science
into great medicine
for rare genetic diseases.

Deep and diversified product pipeline

Developing multiple clinical-stage programs in parallel                                  

Focused on serious rare and ultra-rare diseases

Working with patients and medical professionals to treat rare diseases

Dedicated to creating new treatments 

Join a high-performance team focused on rare diseases

January 4, 2019
Ultragenyx Announces Positive Topline Results from First Cohort of Phase 1/2 Clinical Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia

January 3, 2019
Ultragenyx to Present at J.P. Morgan Healthcare Conference

December 6, 2018
Ultragenyx and Kyowa Kirin Announce Health Canada Approval of Crysvita™ (burosumab injection) for the Treatment of X–linked Hypophosphatemia (XLH) in Adults and Children

  • About
    • Management
      • Emil D. Kakkis, M.D., Ph.D.
      • Camille Bedrosian, M.D.
      • Wladimir Hogenhuis, 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 TIO
    • UX007 for FAOD
    • 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
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    • Events & Presentations
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      • Management
      • Board of Directors
      • Committee Composition
    • SEC Filings
    • Stock Information
      • Analyst Coverage
    • Investor FAQ
    • Contact Us
  • Careers
    • Commitment
    • Benefits
    • Interns
    • Jobs
  • Partnering
    • Current Partnerships
  • Contact
    • Driving Directions
  • Home
  • About
    • Management
      • Emil D. Kakkis, M.D., Ph.D.
      • Camille Bedrosian, M.D.
      • Wladimir Hogenhuis, 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 TIO
    • UX007 for FAOD
    • 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
    • Press Releases
    • Events & Presentations
    • Corporate Governance
      • Management
      • Board of Directors
      • Committee Composition
    • SEC Filings
    • Stock Information
      • Analyst Coverage
    • Investor FAQ
    • Contact Us
  • Careers
    • Commitment
    • Benefits
    • Interns
    • Jobs
  • Partnering
    • Current Partnerships
  • Contact
    • Driving Directions
  • Privacy Policy
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