Vamshi K. Rao

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

<i>EPG5</i> -related Vici syndrome: a paradigm of neurodevelopmental disorders with defective autophagy

Susan Byrne, Jean-Marie U-King-Im, Ata Siddiqui et al. · Brain · 2016 · 138 citations · Full text

Comparative Proteomes of the Proliferating C2C12 Myoblasts and Fully Differentiated Myotubes Reveal the Complexity of the Skeletal Muscle Differentiation Program

Nilesh S. Tannu, Vamshi K. Rao, Ritcha M. Chaudhary et al. · Molecular & Cellular Proteomics · 2004 · 97 citations · Full text

Mouse Survival Motor Neuron Alleles That Mimic SMN2 Splicing and Are Inducible Rescue Embryonic Lethality Early in Development but Not Late

Suzan M. Hammond, Rocky G. Gogliotti, Vamshi K. Rao et al. · PLoS ONE · 2010 · 80 citations · Full text

Combination molecular therapies for type 1 spinal muscular atrophy

Yohei Harada, Vamshi K. Rao, Kapil Arya et al. · Muscle & Nerve · 2020 · 78 citations