- Rare diseases are often chronic, progressive,degenerative, and often life-threatening;
- Rare diseases are disabling: the quality of life of patients is often compromised by the lack or loss of autonomy;
- High level of pain and suffering for the patient and his/her family;
- No existing effective cure;
- There are between 6,000 and 8,000 rare diseases;
- 75% of rare diseases affect children;
- 30% of rare disease patients die before the age of 5;
- 80% of rare diseases have identified genetic origins;
- Other rare diseases are the result of infections (bacterial or viral), allergies and environment.
Characteristics of Rare Diseases
