Rare diseases (orphan diseases) - diseases that affect a small part of the population. State support is usually needed to stimulate their research and develop drugs. Many rare diseases are genetic, and therefore accompany a person throughout life, even if the symptoms do not appear immediately.
Many rare diseases occur in childhood, and about 30% of children with rare diseases do not live to 5 years. There is no single level of prevalence in the population in which it is beginning to be considered rare. The disease may be rare in one part of the world or among a group of people, but is common in other regions or among other groups of people.
In Ufonda, we have included in the program of Orphan diseases, seven of the most common rare diseases among Ukrainians:
1. Bullous epidermolysis
2. Imperfect osteogenesis
3. Cystic fibrosis
4. Mucopolysaccharidosis
5. Gaucher disease
6. Pulmonary (arterial) (arterial) hypertension (idiopathic, periatic,
7. Primary (congenital) immunodeficiency (with predominant antibody deficiency or severe combined immunodeficiency)