Support Rare Disease Communities

Join us in improving the health and well‑being of people living with rare diseases by driving advances in care, research and policy.

Donate Now

About Us

Our organization is dedicated to the rare disease community. Our mission is to improve the health and well‑being of people with rare diseases by driving advances in care, research and policy. We provide trusted information, resources and support for patients and families, while working with researchers, clinicians and policymakers to accelerate progress.

We believe that every person living with a rare condition deserves hope and a fulfilling life. Through advocacy, education and collaborative research, we strive to shorten the diagnostic odyssey, improve access to treatments and strengthen the voice of the rare disease community.

Abstract infographic illustrating rare disease statistics

Our Work

Advocacy & Policy

We advocate for policies that improve access to diagnosis, treatment and care for individuals with rare diseases. Our policy team works with lawmakers to pass legislation that supports research funding, insurance coverage and patient protections.

Research & Innovation

We collaborate with scientists and clinicians to drive research into the causes, treatments and cures of rare diseases. By supporting collaborative studies and sharing data, we accelerate discovery and translate findings into new therapies.

Patient Support

Our assistance programs help patients and families navigate a rare diagnosis, access financial assistance and connect with communities experiencing similar challenges. We offer educational resources and compassionate guidance at every step.

Education & Awareness

Through webinars, conferences and outreach campaigns we raise awareness about rare diseases among healthcare providers and the public. We empower patients to become advocates and educate communities about the importance of early diagnosis.

Our Impact

Rare diseases are individually uncommon, but collectively they affect millions of people. Nearly 1 in 10 Americans suffer from a rare disease, equating to more than 25 million people. Many rare diseases affect children, and a significant proportion of children with rare conditions will not survive past their fifth birthday. There are thousands of rare conditions and together they form a major public health challenge.

7,000+

Estimated number of rare diseases

25M+

Americans living with rare diseases

10%

Approximate proportion of the population impacted

50%

Patients who are children

Get Involved

Volunteer

Join our volunteer network and support outreach, advocacy or event planning. Your time and skills can make a huge difference in the lives of families dealing with rare diseases.

Fundraise

Organize a community fundraiser, run a marathon or host a bake sale. Fundraising helps us expand programs and provide direct assistance to those in need.

Share Your Story

Your personal journey with a rare condition can inspire others and raise awareness. Share your story to give hope and insight to patients, caregivers and policymakers.

Donate

Financial support powers our mission. Whether a one‑time gift or a recurring contribution, your generosity helps drive research, advocacy and support services.