Latin American geneticists have been contributing to the scientific development of Human and Medical Genetics fields since the early 1950s. In the last decades, as Medical Genetics is moving toward a new era of innovative therapies for previously untreatable conditions, the participation of Latin America in clinical trials is also increasing. This review discusses the particularities regarding funding, regulatory, and ethical aspects of conducting clinical trials for genetic diseases in Latin America, with an especial focus on Brazil, the largest country with the highest number of studies. Although there are still several barriers to overcome, the recent development of orphan drug legislation and policies for rare diseases in many Latin American countries indicates a growing opportunity for the participation of the region in international efforts for the development of new therapies for genetic diseases.

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拉丁美洲遗传病临床试验

自 1950 年代初以来，拉丁美洲遗传学家一直为人类和医学遗传学领域的科学发展做出贡献。在过去的几十年里，随着医学遗传学正朝着针对以前无法治愈的疾病的创新疗法的新时代迈进，拉丁美洲参与临床试验的人数也在增加。本综述讨论了在拉丁美洲进行遗传疾病临床试验的资金、监管和伦理方面的特殊性，特别关注巴西这个研究数量最多的最大国家。尽管仍有许多障碍需要克服，