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
Diseases
80%
Genetic
Origin
Origin
95%
NO FDA
Approved Drug
Approved Drug
50%
Affected
are Children
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