P. Shieh

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.

Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study

R. Finkel, M. Cudkowicz, S. Rutkove, 2016, Annals of clinical and translational neurology.