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Gene therapy for selected neuromuscular and trinucleotide repeat disorders – An insight to subsume South Asia for multicenter clinical trials

BACKGROUND: In this article, the authors discuss how they utilized the genetic mutation data in Sri Lankan Duchenne muscular dystrophy (DMD), Spinal muscular atrophy (SMA), Spinocerebellar ataxia (SCA) and Huntington’s disease (HD) patients and compare the available literature from South Asian count...

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Detalles Bibliográficos
Autores principales: Wijekoon, Nalaka, Gonawala, Lakmal, Ratnayake, Pyara, Sirisena, Darshana, Gunasekara, Harsha, Dissanayake, Athula, Senanayake, Sunethra, Keshavaraj, Ajantha, Hathout, Yetrib, Steinbusch, Harry W.M., Mohan, Chandra, Dalal, Ashwin, Hoffman, Eric, D de Silva, K.Ranil
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9931913/
https://www.ncbi.nlm.nih.gov/pubmed/36819775
http://dx.doi.org/10.1016/j.ibneur.2023.01.009