Other Rare Disease Organizations




National Organization For Rare Disorders

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.

Global Genes 

Global Genes provides hope for the more than 400 million people affected by rare disease around the globe. Its mission is to  help patients find and build communities, gain access to information and resources, connect to researchers, clinicians, industry, government, and other stakeholders, share data and experiences, stand up, stand out, and become effective advocates on their own behalf.

EveryLife Foundation 

The EveryLife Foundation for Rare Diseases is a 501(c)(3) nonprofit, nonpartisan organization dedicated to empowering the rare disease patient community to advocate for impactful, science-driven legislation and policy that advances the equitable development of and access to lifesaving diagnoses, treatments and cures.

The Orphan Disease Center At The University of Pennsylvania 

The mission of the Orphan Disease Center will develop transformative therapies using platform technologies that can be deployed across multiple rare diseases. We will emphasize disorders with substantial unmet need independent of their incidence and will strive to assure access to patients of all populations.

The National PKU Alliance

The National PKU Alliance works to improve the lives of families and individuals associated with PKU through research, support, education and advocacy, while ultimately seeking a cure. The NPKUA was formed in 2008 by parents, grandparents, and individuals across the country just like you. We are the first national non-profit organization to unite adults, families, regional and statewide PKU organizations, the medical community, and industry partners to make a difference in the lives of people with PKU.


 Genetic Alliance 

Genetic Alliance engages individuals, families and communities to transform health. This requires that we as an organization understand what it means to transform systems, dissolve boundaries, create and sustain open space, and promote the process of openness. We understand that to transform systems, we must start with our own, beginning with our very person. Such work calls us, both personally and professionally, to explore the deepest truths about ourselves and the systems we impact.