Clinical trials for genetic diseases in Latin America. Issue 3 (3rd September 2021)

• Record Type: Journal Article
• Authors: Poswar, Fabiano de Oliveira; da Silva, Larissa Pozzebon; Zambrano, Marina Bauer; Pedrini, Diane Bressan; Saute, Jonas Alex Morales; Giugliani, Roberto
• Other Names: Prada Carlos E guestEditor.; Schwartz Ida guestEditor.; Cavalcanti Denise guestEditor.
• Is Part Of: American journal of medical genetics. Volume 187:Issue 3(2021)
• Page Start: 381
• Page End: 387
• Publication Date: 2021-09-03
• Subjects: bioethics -- clinical trials -- drug development -- medical genetics -- rare diseases; Medical genetics -- Periodicals; 616.04205
• DOI: 10.1002/ajmg.c.31934
• Languages: English
• ISSNs: 1552-4868

Abstract

Abstract: 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.