Jun Lee

Fukutin mutations in congenital muscular dystrophies with defective glycosylation of dystroglycan in Korea

C. Ki, W. Park, Jong-Won Kim, 2010, Neuromuscular Disorders.

INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular Myopathy

F. Muntoni, L. Servais, G. Rafferty, 2022, Journal of neuromuscular diseases.

Estimation of the Quality-of-Life Impact of X-Linked Myotubular Myopathy

A. Beggs, A. Lloyd, D. Bilder, 2021, Journal of neuromuscular diseases.

ASPIRO Gene Therapy Trial In X-Linked Myotubular Myopathy (XLMTM): Update on Preliminary Safety And Efficacy Findings up to 72 Weeks Post-Treatment (1053)

F. Muntoni, L. Servais, J. Dowling, 2020 .

Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial

A. Buj-Bello, Jun Lee, E. James, 2023, The Lancet Neurology.