Rare Diseases and Orphan Drugs

Biography

Biography: Jorge-Alberto Ascencio-Aragón

Abstract

Chagas disease or American trypanosomiasis is recognized by the World Health Organization as one of the 18 Neglected Tropical Diseases. It is estimated that there are between 7–8 million people infected, between 65–100 million people at risk of becoming infected and it causes nearly 12,000 deaths per year worldwide. Chagas disease is endemic of 21 Latin American countries, but the expanding migrational flows have male the disease an international health priority. It is a parasitic zoonosis caused by Trypanosoma cruzi, a protozoan with high genetic and phenotypic diversity that can be principally transmitted to human beings by the faeces of blood-sucking triatomines. Other mechanisms of transmission include; blood transfusions, organ or bone marrow transplants, from mother to child, by ingestion of food or drinks contaminated with triatomine faeces and due to occupational exposure. Chagas disease has a very broad-spectrum of clinical manifestations, depending upon the phase at where the patient is. Acute phase is characterized for passing unnoticed in 95% of the cases, unless Romaña’s sign or chagoma develops at the inoculation site. Chronic phase is characterized for developing cardiac or gastrointestinal disease that lead to increased morbi-mortality. Diagnosis can be done with the combination of epidemiological background and clinical manifestations, if present, but laboratory tests are required for confirmation. Benznidazole and Nifurtimox are the only drugs available for treating the disease and meanwhile the efforts to formulate vaccines remain insufficient, patients suffer from a preventable disease which main risk factor for acquiring it is living in poor and marginalised societies. Control of vector-borne transmission remains to be a challenge in endemic countries as it is related to low socioeconomic status, while serological screening at blood banks and monitoring of all pregnant women for non vector-borne transmission can be effective to control the disease in non-endemic countries