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Management of neuromuscular diseases and spinal muscular atrophy in Latin America

Gene Therapy volume 24, pages 578–580 (2017)Cite this article

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Abstract

Latin America (LA) has a population of ~645 million people distributed over 33 countries with marked political, cultural and economic differences. In LA, patients with inherited neuromuscular diseases (NMDs) often do not have access to specialized medical centers and many of them go undiagnosed. General management and care of spinal muscular atrophy (SMA) patients in the region varies due to heterogeneous health care. An active generation of young clinical neurologists is being trained for the specialized care of SMA and other neuromuscular (NM) patients, both in the private and public sectors. The Euro-Latin-American Summer School of Myology (EVELAM) as well as efforts of professionals at large public centers in the major cities of LA have a leading role in this development. Different regional academic–scientific organizations as well as the expanding number of telethon centers and the creation of parent organizations, mostly concerning SMA, all together are contributing to the increased quality of the management of NMD patients. Over the past years, academic and clinical research, as well as the establishment of qualified centers for the molecular testing of NMD are pushing forward the creation of patient registries and the development of specific clinical trials, with Argentina and Brazil having a major role in this field. Nevertheless, increased awareness and further training of specialized health professionals are necessary to reach patients that are currently lacking care throughout the region.

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Acknowledgements

We thank patients and their families for their support and Dr Roberto Caraballo for the critical reading of the manuscript. ALR is Principal Investigator from CONICET. Work in the laboratory of ALR is supported by grants from CONICET, FONCYT and The FSH Society grant FSHS-82015-03.

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Authors and Affiliations

  1. Servicio de Neurología, Hospital Nacional de Pediatría Juan P, Buenos Aires, Argentina

    S Monges

  2. Fundacion Allende y Sanatorio Allende, Córdoba, Argentina

    A L Rosa

  3. IRNASUS-CONICET, Universidad Católica de Córdoba, Córdoba, Argentina

    A L Rosa

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  1. S Monges
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Correspondence to S Monges or A L Rosa.

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Monges, S., Rosa, A. Management of neuromuscular diseases and spinal muscular atrophy in Latin America. Gene Ther 24, 578–580 (2017). https://doi.org/10.1038/gt.2017.68

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