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LEADING THE FUTURE OF RARE DISEASE MEDICINE FOR PATIENTS LIKE MASON.

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If you have any difficulty using our website and need an accommodation due to a
disability, you may contact us at: (888) 384-0599

We use our experience, insight, and commitment to move the rare disease
community forward.

Our goal is to provide medicines to those with limited options and to help
patients face rare diseases head on, with courage and confidence.

Press Releases

Ultragenyx has acquired GeneTx to continue to advance development of GTX-102 for
Angelman Syndrome. You can read more

here.
Pipeline

Our diverse portfolio of approved medicines and investigational therapies is
advancing first- or best-in-class treatments for diseases that exhibit a clear
biology for treatment and are severely underserved by current standard of care.

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Culture

We maintain an environment that puts people first. This includes patients, their
families and caregivers, and our employees. We respect the responsibility that
comes with developing medicines for rare diseases.

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LATEST ANNOUNCEMENTS

Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)

October 4, 2022

Ultragenyx and Mereo BioPharma to Present Setrusumab Data Update at ASBMR

September 9, 2022

Ultragenyx to Participate at Citi BioPharma Conference

September 1, 2022

Read More News


COVID-19 INFORMATION

As the COVID-19 pandemic continues to evolve, we are focused on the health and
safety of our community including patients, caregivers, healthcare providers,
partners, and employees. For patients specifically, this means that we are
committed to providing an uninterrupted supply of our medicines to the patients
who rely on them.

Learn More

 * Our Purpose
   * Our Commitment to Patients
   * Supporting Access for Patients
   * Corporate Sustainability
 * Our Company
   * Our Leaders
   * Our Partners
   * Contact Us
 * Our Medicines
   * Crysvita® (burosumab-twza)
   * Dojolvi® (triheptanoin)
   * Mepsevii®(vestronidase alfa—vjbk)
 * Our Research
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Our Other Websites
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