Ultragenyx works side by side with many patient organizations that provide educational resources and support research to help people affected by rare and serious disorders. The organizations listed on this page provide support and information that are helpful across many types of diseases. They are often called “umbrella” organizations. For disease-specific advocacy organizations or information, see here.

Patient Organizations*

Global Genes

Global Genes is a leading rare disease advocacy organization. Its mission is to build awareness, educate the global community, and provide critical connections and resources that empower advocates to become activists for their disease.

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EURORDIS

The mission of EURORDIS is to build a strong pan-European community of patient organizations and people living with rare diseases, to be their voice at the European level, and – directly or indirectly – to fight against the impact of rare diseases on their lives.

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NORD logo

National Organization for Rare Disorders (NORD)

NORD is a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them. NORD, along with its more than 300 patient organization members, is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services.

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CORD logo

Canadian Organization for Rare Disorders (CORD)

CORD is Canada’s national network for organizations representing all those with rare disorders. CORD provides a strong common voice to advocate for health policy and a healthcare system that works for those with rare disorders. CORD works with governments, researchers, clinicians and industry to promote research, diagnosis, treatment and services for all rare disorders in Canada.

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Caregiver Action Network

Caregiver Action Network

The Caregiver Action Network is a family caregiver organization working to improve the quality of life for the more than 90 million Americans who care for loved ones with chronic conditions, disabilities, disease, or the frailties of old age. It supports family caregivers through education, information, referral services, and advocacy.

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Everylife Foundation

EveryLife Foundation for Rare Diseases

The EveryLife Foundation for Rare Diseases is dedicated to advancing the development of treatment and diagnostic opportunities for rare disease patients through science-driven public policy. The Foundation does not speak for patients, but instead provides the training, education, resources and opportunities to make patient voices heard.

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MSU poster

Metabolic Support UK

Metabolic Support UK is the leading patient organization for Inherited Metabolic Diseases supporting thousands of patients worldwide with more than 700 Inherited Metabolic Diseases. Its mission is to raise awareness and provide bespoke support to families, patients and professionals to improve the lives of those affected by Inherited Metabolic Diseases.

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RNE

Rare New England (RNE)

Rare New England’s mission is to bring together New England patients, families and providers touched by rare and complex disorders. The organization offers educational opportunities, creates awareness of available resources, and builds foundations for support to improve patient quality of life.

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Danny Dose

Danny’s Dose

The mission of Danny’s Dose is to raise awareness about the current gap in emergency treatment for those with special medical needs, change the current protocols, help educate emergency service personnel, and educate affected families in proper emergency planning and protection.

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PtoP USA logo

Parent to Parent USA

Parent to Parent USA provides emotional & informational support for families of children who have special needs.

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Additional Resources*

Faster Cures

FasterCures: Patients Count

For too long, patients’ unmet medical needs have been an afterthought, rather than a starting point, for developing medical products that address their priorities and deliver value to the health-care system. FasterCures aims to improve health by driving adoption of methods by which patients’ perspectives shape processes for discovering, developing and delivering medical products and services.

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Our Odyssey

Our Odyssey

Our Odyssey connects young adults impacted by a rare or chronic condition with social and emotional support in the hope of improving their quality of life.

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Logo reading The Mighty

The Mighty

The Mighty is a digital health community created to empower and connect people facing health challenges and disabilities.

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Chronically Capable

Chronically Capable

Chronically Capable is an employment platform that connects chronically ill professionals to companies looking to hire a digital workforce and meet the 7% utilization goal required under Section 503 of the Rehabilitation Act.

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RAM logo

Rare Advocacy Movement

The Rare Advocacy Movement (RAM) is a patient advocacy initiative focused on documenting the evolving complex structure and unique dynamics of the rare disease patient advocacy landscape. RAM is a cultural awakening facilitated by dedicated advocacy leaders who have vowed to remain transparent, clarify misunderstandings and to ensure that the rare disease community is not overlooked, ignored or misrepresented.

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*These organizations are an incomplete listing of rare disease support organizations and are not controlled by, endorsed by, or affiliated with Ultragenyx Pharmaceutical Inc. The list is meant for informational purposes only and is not intended to replace your healthcare professional’s medical advice. Ask your doctor or nurse any questions you may have about your disease or treatment plan.