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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Collage of images of people

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

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.

Your Voice Matters: We are Listening

Jonah

Glycogen Storage Disease Type Ia (GSDIa)

LC-FAOD Community Voices

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

Tiffany and Wil

Duchenne Muscular Dystrophy (DMD)

Myriam and Victoria

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

Regina

Creatine Transporter Deficiency (CTD)

Ann

Tumor-induced Osteomalacia (TIO)

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

OI

Osteogenesis Imperfecta (OI)

Wilson Disease

Wilson Disease

GSDIII

Glycogen Storage Disease
Type III

CDKL5 Deficiency

CDKL5 Deficiency

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