To qRare 'cure rare' diseases

Our Globe . Our Mission

Rare diseases cross borders, and so does our commitment to patients globally


~ 400,000,000

Approximately 400 million individuals are affected with rare diseases in the world.

~ 8,000

There are nearly 8,000 rare diseases identified to date.

~ 5%

Only about 5% of all identified rare diseases have at least one approved therapy.

1

Every single patient with a rare disease is our hero for whom we hope to develop an effective therapy.