Edward C. Smith

Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial

John Day, Richard S. Finkel, Claudia A. Chiriboga et al. · The Lancet Neurology · 2021 · 449 citations

Rare Diseases, Spinal Cord Injury, Medicine +5

Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping

Paula R. Clemens, Vamshi K. Rao, Anne M. Connolly et al. · JAMA Neurology · 2020 · 250 citations · Full text

Handbook of Analytical Derivatization Reactions

Edward C. Smith · Journal of AOAC INTERNATIONAL · 1980 · 164 citations · Full text

Derivative (Chemistry), Engineering, Reaction Process +13

Loss of tubulin deglutamylase <scp>CCP</scp> 1 causes infantile‐onset neurodegeneration

Vandana Shashi, Maria M. Magiera, Dennis Klein et al. · The EMBO Journal · 2018 · 122 citations · Full text

Neurodegenerative Diseases, Neurobiology Of Disease, Molecular Neuroscience +7

IRF2BPL Is Associated with Neurological Phenotypes

Paul C. Marcogliese, Vandana Shashi, Rebecca C. Spillmann et al. · The American Journal of Human Genetics · 2018 · 108 citations · Full text

Neurology, Neuroscience, Molecular Neurobiology +4