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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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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Latest Blog Post

Become Part of the Solution: Participate in Clinical Research

Clinical research plays a critical part in advancing science and treatments. Volunteers who participate in this research are essential – new treatments for rare diseases cannot be approved without them.

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.