| Safety, tolerability, and efficacy of viltolarsen in boys with Duchenne muscular dystrophy amenable to exon 53 skipping: a phase 2 randomized clinical trial PR Clemens, VK Rao, AM Connolly, AD Harper, JK Mah, EC Smith, ... JAMA neurology 77 (8), 982-991, 2020 | 217 | 2020 |
| Comparative proteomes of the proliferating C2C12 myoblasts and fully differentiated myotubes reveal the complexity of the skeletal muscle differentiation program NS Tannu, VK Rao, RM Chaudhary, F Giorgianni, AE Saeed, Y Gao, ... Molecular & Cellular Proteomics 3 (11), 1065-1082, 2004 | 116 | 2004 |
| EPG5-related Vici syndrome: a paradigm of neurodevelopmental disorders with defective autophagy S Byrne, L Jansen, JM U-King-Im, A Siddiqui, HGW Lidov, I Bodi, L Smith, ... Brain 139 (3), 765-781, 2016 | 111 | 2016 |
| Mouse survival motor neuron alleles that mimic SMN2 splicing and are inducible rescue embryonic lethality early in development but not late SM Hammond, RG Gogliotti, V Rao, A Beauvais, R Kothary, CJ DiDonato PloS one 5 (12), e15887, 2010 | 84 | 2010 |
| Gene therapy for spinal muscular atrophy: an emerging treatment option for a devastating disease VK Rao, D Kapp, M Schroth Journal of managed care & specialty pharmacy 24 (12-a Suppl), S3-S16, 2018 | 77 | 2018 |
| The care of patients with Duchenne, Becker, and other muscular dystrophies in the COVID‐19 pandemic A Veerapandiyan, KR Wagner, S Apkon, CM McDonald, KD Mathews, ... Muscle & Nerve 62 (1), 41-45, 2020 | 75 | 2020 |
| Combination molecular therapies for type 1 spinal muscular atrophy Y Harada, VK Rao, K Arya, NL Kuntz, CJ DiDonato, ... Muscle & nerve 62 (4), 550-554, 2020 | 69 | 2020 |
| Paraffin-wax-coated plates as matrix-assisted laser desorption/ionization sample support for high-throughput identification of proteins by peptide mass fingerprinting NS Tannu, J Wu, VK Rao, HS Gadgil, MJ Pabst, IC Gerling, R Raghow Analytical biochemistry 327 (2), 222-232, 2004 | 68 | 2004 |
| Spinal muscular atrophy care in the COVID‐19 pandemic era A Veerapandiyan, AM Connolly, RS Finkel, K Arya, KD Mathews, ... Muscle & nerve 62 (1), 46-49, 2020 | 49 | 2020 |
| Long-term functional efficacy and safety of viltolarsen in patients with Duchenne muscular dystrophy PR Clemens, VK Rao, AM Connolly, AD Harper, JK Mah, CM McDonald, ... Journal of neuromuscular diseases 9 (4), 493-501, 2022 | 39 | 2022 |
| An expanded access program of risdiplam for patients with Type 1 or 2 spinal muscular atrophy JM Kwon, K Arya, N Kuntz, HC Phan, C Sieburg, KJ Swoboda, ... Annals of clinical and translational neurology 9 (6), 810-818, 2022 | 21 | 2022 |
| Efficacy and safety of viltolarsen in boys with Duchenne muscular dystrophy: results from the phase 2, open-label, 4-year extension study PR Clemens, VK Rao, AM Connolly, AD Harper, JK Mah, CM McDonald, ... Journal of neuromuscular diseases 10 (3), 439-447, 2023 | 20 | 2023 |
| PIGQ glycosylphosphatidylinositol‐anchored protein deficiency: characterizing the phenotype LJ Starr, JW Spranger, VK Rao, R Lutz, AT Yetman American Journal of Medical Genetics Part A 179 (7), 1270-1275, 2019 | 19 | 2019 |
| Symptomatic cerebral vasospasm following resection of a medulloblastoma in a child VK Rao, A Haridas, TT Nguyen, R Lulla, MS Wainwright, JL Goldstein Neurocritical care 18, 84-88, 2013 | 14 | 2013 |
| Investigators CINRGDNHS (2020) Safety, tolerability, and efficacy of viltolarsen in boys with duchenne muscular dystrophy amenable to exon 53 skipping: a phase 2 randomized … PR Clemens, VK Rao, AM Connolly, AD Harper, JK Mah, EC Smith, ... doi. org/10.1001/jaman eurol, 2020 | 8 | 2020 |
| Spinal muscular atrophy diagnosed by newborn screening M Lopez-Chacon, AN Buehner, VK Rao Pediatric Neurology Briefs 33, 5, 2019 | 8 | 2019 |
| Clinical trial highlights: O. 2 ignite DMD Phase I/II ascending dose study of SGT-001 microdystrophin gene therapy for DMD: 1.5-year functional outcomes update V Rao, B Byrne, P Shieh, S Salabarria, J Berthy, M Corti, S Redican, ... Neuromuscular Disorders 31, S47, 2021 | 6 | 2021 |
| Parent project muscular dystrophy females with dystrophinopathy conference, Orlando, Florida June 26–June 27, 2019 S Apkon, K Kinnett, L Cripe, D Duan, JL Jackson, JN Kornegay, ML Mah, ... Journal of neuromuscular diseases 8 (2), 315, 2021 | 6 | 2021 |
| Efficacy and safety of vamorolone over 48 weeks in boys with Duchenne muscular dystrophy: a randomized controlled trial UJ Dang, JM Damsker, M Guglieri, PR Clemens, SJ Perlman, EC Smith, ... Neurology 102 (5), e208112, 2024 | 5 | 2024 |
| Transforaminal intrathecal access for injection of nusinersen in adult and pediatric patients with spinal muscular atrophy T Shokuhfar, RN Abdalla, MC Hurley, P Nazari, SA Ansari, S Ajroud-Driss, ... Journal of Pediatric Neurology 18 (02), 088-094, 2020 | 5 | 2020 |
