Synapcell supports rare disease day
rare disease day : the fight goes on...
prevalence of rare diseases
A disease is considered as rare when it affects a small percentage of the population, 1 in 2000 people with an estimate of 30 million of people living with a rare disease.
diagnosis : a tortuous path
There are over 6000 distinct rare diseases and 72% are of genetic origin.
Since these diseases have a low prevalence, medical expertise is rare and patients wait in average 5 years to get diagnosis.
the impact of rare diseases on families
Beyond the person diagnosed, the disease has also an impact on families, care takers and the society as whole, more than 2/3 of family carers spend more than 2 hours a day on disease related tasks.
treatment against rare diseases
Nowadays, only 230 treatments are authorized in the EU with the aim at having 1000 new therapies by 2030
the challenges of drug discovery in rare diseases
Developing new therapeutics in rare disorders is a complicated and long path. The main difficulties is to develop and characterise relevant models and coupled them with the right biomarker to identify new therapeutical strategies.