Making a Difference for Patients:

It’s Up to U

At Ultragenyx, we strive to incorporate patient and family voices into everything we do. Watch our video celebrating how U motivate and inspire us every day.

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COVID-19 Resources & Information

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Patient Advocacy in Action

This summer we hosted a Rare Family Day webinar. Check out the recording to hear from some incredible advocates!

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Upcoming Events

  1. Feb 22

    Rare Across America

    Learn More
  2. Feb 28

    Rare Disease Day

    Learn More

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Latest Blog Post

Collecting Rare Disease Data: Why it Matters

Rare diseases affect a relatively small number of people, so it can be difficult for healthcare providers, government agencies, advocacy groups, pharmaceutical companies, and others to access information about the experiences of those living with rare diseases.

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Featured Educational Resources

Your Voice Matters: We are Listening

Mason

Angelman Syndrome (AS)

Colin

Angelman Syndrome (AS)

Marah

X-linked Hypophosphatemia (XLH)

Franco

Creatine Transporter Deficiency (CTD)

Michelle and Jake

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

Braylee

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

Helen

Mucopolysaccharidosis Type VII (MPS VII)

Cheryl

X-linked Hypophosphatemia (XLH)

Tasia

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

Dan

Tumor-induced Osteomalacia (TIO)

David

X-linked Hypophosphatemia (XLH)

Eileen and John

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

OTC

Ornithine Transcarbamylase Deficiency

GSDIa

Glycogen Storage Disease
Type Ia

Wilson Disease

Wilson Disease

GSDIII

Glycogen Storage Disease
Type III

CDKL5 Deficiency

CDKL5 Deficiency

Click here to learn more about Ultragenyx’s response to the evolving COVID-19 pandemic.

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        By providing your information you agree to allow Ultragenyx and its agents to collect the name and email address provided and to be contacted by Ultragenyx and its agents using this information in the future. Furthermore, you are agreeing that Ultragenyx and its agents may contact you by email. Ultragenyx will not sell, rent or otherwise distribute your name and email address provided.