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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


 

October 18, 2018
Ultragenyx Announces Approval of Mepsevii™ (vestronidase alfa) in Brazil for the Treatment of Mucopolysaccharidosis VII

September 27, 2018
Ultragenyx Announces Positive Topline Cohort 2 Results from Phase 1/2 Clinical Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency and Progression to Higher Dose

September 26, 2018
Ultragenyx to Present at Jefferies Gene Therapy Summit

  • 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
    • UX007 for Glut1 DS
    • DTX 301 for OTC
    • DTX 401 for GSDIa
    • Presentations & Publications
  • For Patients
    • XLH
    • TIO
    • MPS 7
    • LC-FAOD
    • Glut1 DS
    • 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
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      • 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
    • UX007 for Glut1 DS
    • DTX 301 for OTC
    • DTX 401 for GSDIa
    • Presentations & Publications
  • For Patients
    • XLH
    • TIO
    • MPS 7
    • LC-FAOD
    • Glut1 DS
    • 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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