| Single-dose gene-replacement therapy for spinal muscular atrophy JR Mendell, S Al-Zaidy, R Shell, WD Arnold, LR Rodino-Klapac, TW Prior, ... New England Journal of Medicine 377 (18), 1713-1722, 2017 | 2154 | 2017 |
| Eteplirsen for the treatment of Duchenne muscular dystrophy JR Mendell, LR Rodino‐Klapac, Z Sahenk, K Roush, L Bird, LP Lowes, ... Annals of neurology 74 (5), 637-647, 2013 | 850 | 2013 |
| Longitudinal effect of eteplirsen versus historical control on ambulation in D uchenne muscular dystrophy JR Mendell, N Goemans, LP Lowes, LN Alfano, K Berry, J Shao, EM Kaye, ... Annals of neurology 79 (2), 257-271, 2016 | 525 | 2016 |
| Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial CM McDonald, C Campbell, RE Torricelli, RS Finkel, KM Flanigan, ... The Lancet 390 (10101), 1489-1498, 2017 | 454 | 2017 |
| Evaluation of postural stability in children: current theories and assessment tools SL Westcott, LP Lowes, PK Richardson Physical therapy 77 (6), 629-645, 1997 | 367 | 1997 |
| 188th ENMC international workshop: inclusion body myositis, 2–4 December 2011, Naarden, The Netherlands MR Rose, enMC iBM Working Group Neuromuscular Disorders 23 (12), 1044-1055, 2013 | 344 | 2013 |
| Assessment of systemic delivery of rAAVrh74. MHCK7. micro-dystrophin in children with Duchenne muscular dystrophy: a nonrandomized controlled trial JR Mendell, Z Sahenk, K Lehman, C Nease, LP Lowes, NF Miller, ... JAMA neurology 77 (9), 1122-1131, 2020 | 294 | 2020 |
| A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy JR Mendell, Z Sahenk, V Malik, AM Gomez, KM Flanigan, LP Lowes, ... Molecular Therapy 23 (1), 192-201, 2015 | 282 | 2015 |
| Five-year extension results of the phase 1 START trial of onasemnogene abeparvovec in spinal muscular atrophy JR Mendell, SA Al-Zaidy, KJ Lehman, M McColly, LP Lowes, LN Alfano, ... JAMA neurology 78 (7), 834-841, 2021 | 217 | 2021 |
| Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy S Al‐Zaidy, AS Pickard, K Kotha, LN Alfano, L Lowes, G Paul, K Church, ... Pediatric pulmonology 54 (2), 179-185, 2019 | 202 | 2019 |
| Impact of age and motor function in a phase 1/2A study of infants with SMA type 1 receiving single-dose gene replacement therapy LP Lowes, LN Alfano, WD Arnold, R Shell, TW Prior, M McColly, ... Pediatric neurology 98, 39-45, 2019 | 183 | 2019 |
| AVXS-101 (onasemnogene abeparvovec) for SMA1: comparative study with a prospective natural history cohort SA Al-Zaidy, SJ Kolb, L Lowes, LN Alfano, R Shell, KR Church, ... Journal of neuromuscular diseases 6 (3), 307-317, 2019 | 167 | 2019 |
| Prospective and longitudinal natural history study of patients with Type 2 and 3 spinal muscular atrophy: Baseline data NatHis-SMA study A Chabanon, AM Seferian, A Daron, Y Péréon, C Cances, C Vuillerot, ... PLoS One 13 (7), e0201004, 2018 | 151 | 2018 |
| Follistatin gene therapy for sporadic inclusion body myositis improves functional outcomes JR Mendell, Z Sahenk, S Al-Zaidy, LR Rodino-Klapac, LP Lowes, ... Molecular Therapy 25 (4), 870-879, 2017 | 124 | 2017 |
| Validity of the Peabody Developmental Gross Motor Scale as an evaluative measure of infants receiving physical therapy RJ Palisano, TH Kolobe, SM Haley, L Pax Lowes, SL Jones Physical therapy 75 (11), 939-948, 1995 | 123 | 1995 |
| Eteplirsen Study Group and Telethon Foundation DMD Italian Network. Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy JR Mendell, N Goemans, LP Lowes, LN Alfano, K Berry, J Shao, EM Kaye, ... Ann. Neurol 79 (2), 257-271, 2016 | 115 | 2016 |
| Gene therapy for spinal muscular atrophy: safety and early outcomes MA Waldrop, C Karingada, MA Storey, B Powers, MA Iammarino, ... Pediatrics 146 (3), 2020 | 114 | 2020 |
| Motor and cognitive assessment of infants and young boys with Duchenne Muscular Dystrophy: results from the Muscular Dystrophy Association DMD Clinical Research Network AM Connolly, JM Florence, MM Cradock, EC Malkus, JR Schierbecker, ... Neuromuscular Disorders 23 (7), 529-539, 2013 | 112 | 2013 |
| Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy LN Alfano, JS Charleston, AM Connolly, L Cripe, C Donoghue, R Dracker, ... Medicine 98 (26), e15858, 2019 | 85 | 2019 |
| Outcome reliability in non‐ambulatory boys/men with Duchenne muscular dystrophy AM Connolly, EC Malkus, JR Mendell, KM Flanigan, JP Miller, ... Muscle & Nerve 51 (4), 522-532, 2015 | 85 | 2015 |
Louise Rodino-KlapacCenter for Gene Therapy-Nationwide Children's Hospital在 nationwidechildrens.org 的电子邮件经过验证
