| 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 | 2251 | 2017 |
| Current clinical applications of in vivo gene therapy with AAVs JR Mendell, SA Al-Zaidy, LR Rodino-Klapac, K Goodspeed, SJ Gray, ... Molecular Therapy 29 (2), 464-488, 2021 | 566 | 2021 |
| 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 | 533 | 2016 |
| 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 | 308 | 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 | 290 | 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 | 241 | 2021 |
| From clinical trials to clinical practice: practical considerations for gene replacement therapy in SMA type 1 SA Al-Zaidy, JR Mendell Pediatric neurology 100, 3-11, 2019 | 216 | 2019 |
| 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 | 213 | 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 | 192 | 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 | 173 | 2019 |
| 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 | 131 | 2017 |
| Follistatin gene therapy improves ambulation in Becker muscular dystrophy SA Al-Zaidy, Z Sahenk, LR Rodino-Klapac, B Kaspar, JR Mendell Journal of neuromuscular diseases 2 (3), 185-192, 2015 | 70 | 2015 |
| Neurological Complications of PCR-Proven M. pneumoniae Infections in Children: Prodromal Illness Duration May Reflect Pathogenetic Mechanism SA Al-Zaidy, D MacGregor, S Mahant, SE Richardson, A Bitnun Clinical Infectious Diseases 61 (7), 1092-1098, 2015 | 68 | 2015 |
| Gene therapy for muscular dystrophy: moving the field forward S Al-Zaidy, L Rodino-Klapac, JR Mendell Pediatric neurology 51 (5), 607-618, 2014 | 54 | 2014 |
| Gene delivery for limb-girdle muscular dystrophy type 2D by isolated limb infusion JR Mendell, LG Chicoine, SA Al-Zaidy, Z Sahenk, K Lehman, L Lowes, ... Human Gene Therapy 30 (7), 794-801, 2019 | 49 | 2019 |
| Clinical follow‐up for Duchenne muscular dystrophy newborn screening: A proposal JM Kwon, HZ Abdel‐Hamid, SA Al‐Zaidy, JR Mendell, A Kennedy, ... Muscle & nerve 54 (2), 186-191, 2016 | 27 | 2016 |
| Duchenne and Becker muscular dystrophy carriers: Evidence of cardiomyopathy by exercise and cardiac MRI testing ML Mah, L Cripe, MK Slawinski, SA Al-Zaidy, E Camino, KJ Lehman, ... International Journal of Cardiology 316, 257-265, 2020 | 23 | 2020 |
| 480. Gene therapy for spinal muscular atrophy type 1 shows potential to improve survival and motor functional outcomes JR Mendell, S Al-Zaidy, R Shell, WD Arnold, L Rodino-Klapac, JT Kissel, ... Molecular Therapy 24, S190, 2016 | 23 | 2016 |
| A roadmap to newborn screening for Duchenne muscular dystrophy SA Al-Zaidy, M Lloyd-Puryear, A Kennedy, V Lopez, JR Mendell International journal of neonatal screening 3 (2), 8, 2017 | 18 | 2017 |
| ACTIVE (Ability Captured Through Interactive Video Evaluation) workspace volume video game to quantify meaningful change in spinal muscular atrophy LN Alfano, NF Miller, MA Iammarino, M Moore Clingenpeel, SL Lowes, ... Developmental Medicine & Child Neurology 62 (3), 303-309, 2020 | 15 | 2020 |
