On May 14, 2020, the Children Health School, a new project launched by the Russian Health Ministry’s National Medical and Research Center for Children’s Health (federal state-owned autonomous institution), was held online.
The advanced research based on the contemporary approach to the key pediatric problems underlies the School’s curriculum. The target audience is pediatricians, allergologists and immunologists, dietologists, neurologists, neonatologists.
NANOLEK supported the session held by Prof. Ludmila Mikhailovna Kuzankova, Ph.D., who covered the Hunter’s Syndrome Diagnosis and Treatment in Children. Ludmila Mikhailovna dwelled on the difficulties with early diagnosis of this rare but severe genetic condition (Type II mucopolysaccharidosis), reviewed the clinical pattern and the multi-disciplinary treatment principles. The second report part presented by Tatiana Vladimirovna Podkletnova, Candidate in Medicine, was devoted to the enzyme substitution treatment of Type II mucopolysaccharidosis and included a clinical case of Idursulphase Beta (Hunterase) administration.
NANOLEK has localized manufacturing of several socially significant drugs that did not use to be produced in Russia, in particular, the original drug of Idursulphase Beta, for Type II mucopolysaccharidosis treatment. The Federal Register of Patients with Life Threatening and Orphan Diseases contains data on 128 patients suffering from Type II mucopolysaccharidosis, as of January 2020, according to the Russian Health Ministry.
Experts have already estimated that the use of original drug of Idursulphase Beta helps save almost RUB 800 million of federal budget funds as compared with the alternative treatment options.