Rare (or orphan) diseases in modern medicine stand apart from such large-scale problems as cardiac, oncological or infectious diseases. It is the low prevalence in the population that is the main reason for the difficulty of their treatment.
The development and introduction into practice of a new medicinal product is a long and expensive process. Therefore, manufacturers always first evaluate the economic feasibility of development. The small number of patients and, accordingly, the non-massive need for drugs for orphan diseases make the search for effective therapeutic products “uninteresting” for pharmaceutical companies.
We deliberately chose orphan diseases as one of our focuses, because every person has the right to a quality life. YURіА-PHARM develops and makes available medicinal products for the treatment of cystic fibrosis (both symptomatic and disease-modifying), primary pulmonary hypertension, idiopathic pulmonary fibrosis, and others. We pay particular attention to the search for mechanisms to improve the availability of these drugs for patients in the context of a shortage of public funding for orphan diseases. For this purpose, active cooperation is conducted with charitable foundations, patient organizations, and the international scientific community is involved.
However, the treatment or therapy of orphan diseases is often not possible using only drugs based on low molecular weight compounds. It requires a targeted approach, for which biotechnological products are used. YURI-PHARM is working on the development of monoclonal antibody preparations, as well as variants of mRNA-ARI for the therapy of rare genetic diseases.