Treatments for rare diseases
Understanding rare diseases
In Europe, a disease is considered rare when it affects fewer than 1 in 2,000 people.
Rare diseases are often complex conditions whose management can be hampered by a lack of knowledge, sometimes late diagnoses and limited treatment options.
Behind the term “rare diseases” lies a reality that affects millions of people around the world. While each disease affects a limited number of patients, their collective impact is considerable, both human and societal.
At KÔL, we are committed to facilitating access to innovative treatments for patients facing these conditions, when treatment options are insufficient or non-existent.
A RARE DISEASE MEANS:
A diagnosis that is often long and complex
Scientific knowledge that is sometimes limited
A lack of available treatment options
A major impact on the daily lives of patients and their families
8,000
Rare diseases identified. More than 7,000 different rare diseases have been described to date, the vast majority of which remain little known.
Source: European Commission
patients in the European Union.
Across Europe, between 27 and 36 million people live with a rare disease.
Source: European Commission
of rare diseases begin in childhood. Most appear in the first years of life, often with serious consequences for patients and their families.
Source: European Commission
have no curative treatment.
Despite advances in research, the vast majority of rare diseases still have no treatment that can cure them.
Source: European Commission
are genetic in origin. Most rare diseases are linked to a genetic abnormality, although their mechanisms are still poorly understood for many of them.