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...

Full description

Bibliographic Details
Main Authors: Nalaka Wijekoon, Lakmal Gonawala, Pyara Ratnayake, Darshana Sirisena, Harsha Gunasekara, Athula Dissanayake, Sunethra Senanayake, Ajantha Keshavaraj, Yetrib Hathout, Harry W.M. Steinbusch, Chandra Mohan, Ashwin Dalal, Eric Hoffman, K.Ranil D de Silva
Format: Article
Language:English
Published: Elsevier 2023-06-01
Series:IBRO Neuroscience Reports
Subjects:
Online Access:http://www.sciencedirect.com/science/article/pii/S266724212300009X