Advocacy

About
rare diseases

A Neglected Emergency

Rare diseases* affect 350 million people globally

Almost 5% of the world

30 million in the USA

Rare diseases are mostly genetic, affect children and are mostly neglected by private and public investors

7,000
Rare
Diseases
80%
Genetic
Origin
95%
NO FDA
Approved Drug
50%
Affected
are Children



Rare diseases affect more people than the AIDS and Cancer communities combined

1 in 10 Americans
1 in 20 Children

*In the US a disease is defined RARE if it affects less than 200,000 people (FDA)
Slides’ Source: NIH

Did you know...

10 CHILDREN in the world are born with a genetic disease EVERY MINUTE
70% OF CHILDREN affected by a genetic disease show symptoms BY AGE 3
Rare diseases are responsible for 35% OF DEATHS IN THE FIRST YEAR OF LIFE
30% OF CHILDREN affected by a rare genetic disease won’t make their 5TH BIRTHDAY