True / False on orphan diseases..

Orphan diseases plague the lives of three million French. Yet we are all concerned. Are you aware of the main challenges?

An orphan disease is rare.

TRUE. An orphan disease affects a small number of people compared to the general population. The threshold is accepted in Europe on a person with 2000, less than 30,000 people for a given disease in France.

Few people are affected by this problem.

FALSE. More than 3 million French people suffer from a rare disease. In Europe, 27 million people are victims of an orphan disease. And 6-8% of the world population would be concerned, directly or indirectly, by these diseases.

There are hundreds of rare diseases identified.

FALSE. Nearly 7,000 rare diseases identified are more or less disabling unknown and neglected. And five new diseases are described every week in the world.
Fifty rare diseases affect each a few thousand people in France, the most famous being cystic fibrosis with 5 000-6 000 patients in France.
Five hundred others did reach a few hundred, as progeria or premature aging that affects only 100 cases worldwide. And thousands of others affect only a few dozen people.

These rare diseases are serious.

TRUE to 65%. Approximately 80% of diseases have an impact on life expectancy. And 35% are directly involved in death before the age of one year. They cause significant discomfort in everyday life in over 65% of cases. They are causing a complete loss of autonomy in 9% of cases.

There are few treatments.
FALSE. Or almost zero treatment currently exists.