Oligofastx is a project born from a need: to offer a therapeutic pathway based on the development of specific oligonucleotides for certain rare diseases for which there is no solution at the moment.
I feel this is our leit-motiv and sign of identity, we could not miss, of course, to join the celebration of Rare Disease Day, which has been celebrated on the last day of February since 2008.
The aim of the commemoration is to raise awareness and help all people suffering from any of the so-called rare diseases, to receive timely diagnosis and treatment and, in the long run, this will guarantee them a better life.
The date of celebration is not a coincidence: the month of February has a very particular characteristic and, depending on the year, it may or may not be a leap year. For this reason, this date was chosen as a symbolic way of associating this rarity with the disease.
So-called rare diseases (RD) are diseases that affect few people compared to the general population and, because of their low frequency, raise special issues both in terms of their social and clinical treatment and in terms of their research
In the European Union, a disease is considered rare if it affects 5 in 100,000 people (1 in 2,000 people). One of the epidemiological characteristics of these diseases is that the highest incidence occurs in the pediatric age group, since a large number of them are due to genetic and congenital causes, while their prevalence is higher in adults due to a very high infant mortality rate for some of them. Their geographic distribution can also vary widely.
Rare diseases are an emerging global public health priority. Given their high heterogeneity and the need to provide individualised medicine for small groups of patients, they have become a challenge for health systems and biopharmaceutical research.
According to available epidemiological data, they affect between 3.5 and 5.9 per cent of the world’s population, equivalent to about 300 million people worldwide, a figure used so far by Rare Diseases International and EURORDIS.
Afforded rare diseases in OLIGOFASTX project are:
- Guillain-Barré syndrome
- Retinitis Pigmentosa
- Leber Congenital Amaurosis
- Cancer cachexia
- Alström Syndrome
- Fuchs’ corneal endothelial dystrophy
- Anaplastic thyroid cancer
Unfortunately, there are many more rare diseases for which there is no solution: some discovered, others yet to be discovered. Therefore, at Oligofastx we want to focus our efforts on these 7 as a priority, and hopefully the projection of the collaboration between the leading Spanish companies that participate in this project will reach much further and allow us to expand our scope of action.