Only 50 of the more than five thousand rare diseases available treatment.
only 50 of the more than five thousand rare diseases has treatment.
February 28 day world the EERR.
– more than 3 million Spaniards suffer any EERR. Approximately 8% of the Spanish population. Although if you consider people who are not diagnosed, the figure rises to 5 million, is one of the problems faced by these patients to late diagnosis, and in many cases erroneous.
– many of these patients have “ a great feeling of abandonment and orphanhoodbecause his illness has a low prevalence of social ”, said the DRA. Ana Villegas.
-Alexion Pharma marketed in 2007 the only treatment approved for patients of PNH, one of the so-called rare diseases, becoming so each of the 50 rare diseases with drug available.
Barcelona, February 2010. It is estimated that there are between five and eight thousand rare diseases. In many cases, sufferers are undiagnosed. Of them only a few approximately fifty have treatment appropriate to his pathology. “ rare diseases are a challenge and are a great clinical complexity at the time of his diagnosis ”, stated DRA. Ana Villegas, hematóloga of the Hospital ClÃnico San Carlos, in the context of the first Bio-talk organized by Alexion Pharma Spain, pioneer in the therapeutic approach of these minority ailments.
“ Have been so long without medication when they get always following the treatment ”, has underlined the DRA. Villegas, who has also reminded “ the importance of research in low-prevalence diseases such ”. The Act also has been Dr. Ãlvaro urban, which has demanded “ the involvement of politicians ” and “ resources to improve the situation of some patients often neglected and without diagnosis ”.
Dr. urban has also highlighted “ the transcendence of the associations of patients especially in such diseases, already that the patient feels alone and thinks that his illness is not socially shared ”. This referred to by Jordi Cruz, President of the Association of patients of PNH, who has insisted on the need that “ both administrations and private companies should follow encouraging research into these diseases, given that for a patient a drug is a light ”. “ How sad it is to have a disease and that there is no treatment for her ”, has been completed.
For its part, the Member of Congress Conchita Turruella has stated that “ administration involves more and more in improving the lives of this kind of sick, but that the work of the associations of patients is very important because they push the Executive to promote legal initiatives such as the research of treatments or psychological assistance to families ”.
Finally, the general director of Alexion Pharma Spain, Jordi Casals, explained that his company sold the only treatment that can improve the quality of life and avoid life-threatening risks to patients of PNH, one of the few rare diseases has drug for your condition.
Rare diseases
Low-frequency diseases and rare diseases (EERR) affects 5 people for every 10,000, according to the classification established by the European Union. This means that the maximum number of patients from a pathology can be considered rare would be 227.000 in the European Union and approximately 22,000 in Spain. In United States, the law provides as EERR one that affects less than 200,000 people.
There are 9,000 different pathologies, according to the latest figures published by the World Health Organization (who).
They are diseases potentially fatal or disabling long-term:
in 50% of the cases in which the vital prognosis is diagnosed an EERR is at risk. Credited for 35% of deaths in children under one year, 10% of deaths between 1 and 5 years and 12% in children aged 5 to 15 years.
65% Of low prevalence diseases are serious and highly disabling. They are characterized by a beginning early in life – 2 of every 3 diseases appear before the two years — they involve chronic pain in 1 of every 5 patients and involve a motor, sensory or intellectual deficit in half the cases.
80% Of cases are congenital (born with them). Also, they usually involve physical malformations or gradual deterioration of motor, sensory and intellectual capacities.
EERR in Spain
More than 3 million Spaniards suffer any EERR. Approximately 8% of the Spanish population. Although if you consider people who are not diagnosed, the figure rises to 5 million, is one of the problems faced by these patients to late diagnosis, and in many cases, wrong.
In all Spain, there are some 500,000 children affected some EERR.
It is estimated that 12% of hospital discharges recorded some EERR diagnosis, which means 445,000 patients each year in Spain.
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