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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

February 21, 2019
Ultragenyx Announces Positive 24-week Data from First Cohort of Phase 1/2 Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia

February 19, 2019
Ultragenyx Reports Fourth Quarter and Full Year 2018 Financial Results and Corporate Update

February 14, 2019
Ultragenyx and Kyowa Kirin Announce Positive 64-Week Results for Crysvita® (burosumab) from Phase 3 Study in Children with X-linked Hypophosphatemia (XLH)

  • 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.
      • Shehnaaz Suliman, M.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
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  • 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.
      • Shehnaaz Suliman, M.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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