Cuts in drugs for rare diseases
HPN Association notes that "health cuts are also being produced, in a disguised manner, in Andalusia, and have begun a very sensitive area and needed as in the Rare Disease."
Paroxysmal nocturnal hemoglobinuria (PNH), a so-called rare diseases, is characterized by destruction of red blood cells and the ability to generate thromboembolic events leading cause of death among these patients. Symptoms include body aches, anemia and blood in urine.
A Josefa twice been denied access to Soliris, the only drug that has shown to improve quality of life of patients with PNH and avoiding any risk of thrombosis, arguing that "does not meet the clinical criteria." According to Jordi Cruz, president of the Association of PNH, "the explanation has no medical basis, and that, in turn, approved the treatment for the patient, but then temporarily suspended for a problem that had encephalitis. It was the pretext that he found the administration to not administer eculizumab, "said Cruz.
Josefa present state is "delicate, is very bad and the worst could happen." Jordi Cruz, "the blockade by the Ministry of Health is due to purely economistic, the cuts have started to produce in the Andalusian health because treatment is expensive, but more expensive can go to eventually play with the health of the people, "concludes George Cross.
In addition, the Association of PNH has been the case to the Andalusian Ombudsman, José Chamizo.
Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra-rare disease that affects about 250 patients in Spain. It is a genetic disorder in which normal natural proteins do not adhere adequately to red blood cells and, consequently, they are vulnerable immune system and are destroyed prematurely and intermittently. A very minor impact disease progressively deteriorating patients and is a real life threatening.
First described in 1882, adopted this name because it was observed that intravascular hemolysis (destruction of red blood cells) occurred in crisis situations and at night, during sleep, after stressful situations, infections, physical exertion, immunizations and acetylsalicylic acid intake.
PNH often unrecognized initially occurs, usually between 30 and 40 years, and the delay in diagnosis may take from 1 to more than 10 years. The value of the median survival for PNH patients is between 10 and 15 years from the time of diagnosis. PNH develops without warning and affects both sexes. Is estimated to affect between 8,000 and 10,000 people in Europe and North America.
Until now, there was no specific therapy for the treatment of PNH. The treatment of this disease is limited to symptom management through periodic blood transfusions, non-specific immunosuppressive therapy and, rarely, bone marrow transplants, a procedure that carries considerable mortality risk.