WHO
We Are
We are advocates, partners and collaborators who are relentless in our efforts to affect change.
WHY
We Do It
To eliminate the daily challenges for 1 in 10 people affected by rare disease.
WHAT
We Do
We empower patients, build communities & drive forward momentum for rare disease globally.
What's New
Gene Editing Treatment Shows Positive Results in First Two Treated Patients with Blood Disorders
Read MoreTogether We Can Change Lives
7000
Distinct Types of Rare and Genetic Diseases
400
Million People Suffer From a Rare Disease Globally (greater than the population of the U.S.)
3of10
Children with a Rare Disease Won’t Live to See Their 5th Birthday
1of 2
Patients Diagnosed with a Rare Disease is a Child
