Although some diseases are rare, the number of people suffering from them is significant. There are between 6,000 and 8,000 rare diseases affecting a total of 6 to 8% of Belgian people.
What are orphan drugs?
Orphan drugs are intended to treat rare diseases.
As the pharmaceutical industries often have less interest in developing medicines for diseases that affect a limited number of people, incentives have been put in place.
They aim among other things to encourage research, development and marketing of new treatments intended to treat rare diseases.
There is no treatment for the majority of rare diseases and certain treatments remain very expensive.
Certain rare metabolic diseases, detected during a neonatal screening test, necessitate an adapted diet for the newborn.
An adapted diet is necessary for all diseases that are detected by neonatal screening.
Research is looking into the possibilities offered by gene therapy.