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On September 26, in the Moscow region, a scientific and practical conference "Rare (orphan) diseases in the practice of a pediatrician" took place, with NANOLEK as a partner.

The problem of treatment orphan diseases is one of the most significant in the whole world, because such diseases are characterized by a low prevalence, and this leads to difficulties in organizing research and production. Starting treatment in the early stages of diagnosis can reduce patient disability and even save his life.

The conference was attended by specialists in rare diseases, as well as endocrinologists, genetics and neurologists; the professional community discussed the modern possibilities of therapy, diagnostic methods, and the organization of medical care for patients with rare diseases.

Professor Bokova Tatyana Alekseyevna, Head of the Orphan Disease Center of the Ministry of Emergencies and Diseases, made a report on the treatment of mucopolysaccharidosis. She described routing, the main symptoms of the disease, and treatment with enzyme replacement therapy, in particular idursulfase beta.

Today, NANOLEK is the one Russian company localizing a drug for the treatment of type 2 mucopolysaccharidosis in the country. In 2018, it was registered and included in the list of Essential Medicines.

The event was organized by the Ministry of Health of the Moscow Region and the Moscow Regional Consultative and Diagnostic Center for Children.