News

On May 12^th-13^th, 2011, “Viva Pharm” company had participated in the International research and practice conference “Problems of diagnosis and treatment of rare (orphan) diseases in children in Kazakhstan”. The purpose of this conference is – improvement of health care delivery for children with rare genetic diseases, such as mucoviscidosis and storage diseases (Gaucher's disease, mucopolysaccharidosis and etc.), the search ways for problem solutions associated with medico-genetic diagnosis, therapy and rehabilitation of patients. Also the problem of medication providing to patients is very critical (especially some medications are not registered in Kazakhstan).

According to the data of Ministry of Public Health on 10.02.11, 39 sick children with mucoviscidosis, 6 patients with Gaucher's disease, 5 patients with mucopolysaccharidosis and 1 child with Pompe disease were registered in the Republic of Kazakhstan. The single pathogenic way of treatment is lifetime substitution treatment with expensive medications (more than 2 000 US dollars in month per one child). Of course none family in our country will not be able to deal with such problem by themselves. In Kazakhstan as in all developed countries of the world this category of patients is provided by medications at the expense of local budgets, but if to take into consideration the high cost of these medications, then these funds are not enough for necessary course of treatment. Therefore, in the purpose of improvement of health care delivery for children, who suffers from rare (orphan) diseases, in the Ministry of Public Health was prepared a Project of Order about appropriation of additional financing with the use of Republican budget. 

Untill this time, medicine has been offering only symptomatic treatment mode of storage diseases, the problem of mucoviscidosis therapy still is not solved totally. Ten years experience of enzyme-substitutive therapy in storage diseases in whole world shows that this treatment mode stops the progression of the disease, promotes regression of symptoms and significantly improves the life quality of patients. Today in the whole world, thousands of patients with Gaucher's disease are getting intravenous injections of modified human glucocerebrosidase. Also at this moment patients with Fabry disease, mucopolysaccharidosis I and II types are receiving specific treatment in abroad; similar methods of correction Pompe disease, mucoviscidosis are in the stage of clinical trials, researches about the possibility of using for this purpose the gene therapy are in progress. 

Due to the success of genetics and modern medical science, the effective treatment of orphan diseases is becoming a reality, reduces pain and sufferings of many people, and gives to the previously hopeless patients a hope for a full life.