| Myostatin inhibitor ACE‐031 treatment of ambulatory boys with Duchenne muscular dystrophy: results of a randomized, placebo‐controlled clinical trial C Campbell, HJ McMillan, JK Mah, M Tarnopolsky, K Selby, T McClure, ... Muscle & nerve 55 (4), 458-464, 2017 | 229 | 2017 |
| Life expectancy at birth in Duchenne muscular dystrophy: a systematic review and meta-analysis E Landfeldt, R Thompson, T Sejersen, HJ McMillan, J Kirschner, ... European journal of epidemiology 35, 643-653, 2020 | 175 | 2020 |
| Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy D Chand, F Mohr, H McMillan, FF Tukov, K Montgomery, A Kleyn, R Sun, ... Journal of hepatology 74 (3), 560-566, 2021 | 168 | 2021 |
| Medical students’ perception of lesbian, gay, bisexual, and transgender (LGBT) discrimination in their learning environment and their self-reported comfort level for caring for … N Nama, P MacPherson, M Sampson, HJ McMillan Medical education online 22 (1), 1368850, 2017 | 156 | 2017 |
| Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial KA Strauss, MA Farrar, F Muntoni, K Saito, JR Mendell, L Servais, ... Nature medicine 28 (7), 1381-1389, 2022 | 142 | 2022 |
| Newborn screening programs for spinal muscular atrophy worldwide: Where we stand and where to go T Dangouloff, E Vrščaj, L Servais, D Osredkar, T Adoukonou, O Aryani, ... Neuromuscular Disorders 31 (6), 574-582, 2021 | 137 | 2021 |
| Emergence of the primary pediatric stroke center: impact of the thrombolysis in pediatric stroke trial TJ Bernard, MJ Rivkin, K Scholz, G deVeber, A Kirton, JC Gill, AK Chan, ... Stroke 45 (7), 2018-2023, 2014 | 130 | 2014 |
| The use of intravenous bisphosphonate therapy to treat vertebral fractures due to osteoporosis among boys with Duchenne muscular dystrophy AM Sbrocchi, F Rauch, P Jacob, A McCormick, HJ McMillan, ... Osteoporosis International 23, 2703-2711, 2012 | 125 | 2012 |
| Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial KA Strauss, MA Farrar, F Muntoni, K Saito, JR Mendell, L Servais, ... Nature medicine 28 (7), 1390-1397, 2022 | 117 | 2022 |
| Serum transaminase levels in boys with Duchenne and Becker muscular dystrophy HJ McMillan, M Gregas, BT Darras, PB Kang Pediatrics 127 (1), e132-e136, 2011 | 114 | 2011 |
| Debunking Occam's razor: Diagnosing multiple genetic diseases in families by whole‐exome sequencing TB Balci, T Hartley, Y Xi, DA Dyment, CL Beaulieu, FP Bernier, L Dupuis, ... Clinical genetics 92 (3), 281-289, 2017 | 108 | 2017 |
| Pan-viral serology implicates enteroviruses in acute flaccid myelitis RD Schubert, IA Hawes, PS Ramachandran, A Ramesh, ED Crawford, ... Nature medicine 25 (11), 1748-1752, 2019 | 96 | 2019 |
| Abnormal fatty acid metabolism is a core component of spinal muscular atrophy MO Deguise, G Baranello, C Mastella, A Beauvais, J Michaud, A Leone, ... Annals of clinical and translational neurology 6 (8), 1519-1532, 2019 | 96 | 2019 |
| Childhood chronic inflammatory demyelinating polyradiculoneuropathy: combined analysis of a large cohort and eleven published series HJ McMillan, PB Kang, HR Jones, BT Darras Neuromuscular Disorders 23 (2), 103-111, 2013 | 84 | 2013 |
| The time to and determinants of first fractures in boys with Duchenne muscular dystrophy J Ma, HJ McMillan, G Karagüzel, C Goodin, J Wasson, MA Matzinger, ... Osteoporosis International 28, 597-608, 2017 | 77 | 2017 |
| Specific combination of compound heterozygous mutations in 17β-hydroxysteroid dehydrogenase type 4 (HSD17B4) defines a new subtype of D-bifunctional … HJ McMillan, T Worthylake, J Schwartzentruber, CC Gottlieb, ... Orphanet journal of rare diseases 7, 1-9, 2012 | 76 | 2012 |
| Developing standardized corticosteroid treatment for Duchenne muscular dystrophy M Guglieri, K Bushby, MP McDermott, KA Hart, R Tawil, WB Martens, ... Contemporary clinical trials 58, 34-39, 2017 | 72 | 2017 |
| Risk of intracranial hemorrhage following intravenous tPA (tissue-type plasminogen activator) for acute stroke is low in children C Amlie-Lefond, DWW Shaw, A Cooper, MS Wainwright, A Kirton, ... Stroke 51 (2), 542-548, 2020 | 70 | 2020 |
| Effect of different corticosteroid dosing regimens on clinical outcomes in boys with Duchenne muscular dystrophy: a randomized clinical trial M Guglieri, K Bushby, MP McDermott, KA Hart, R Tawil, WB Martens, ... Jama 327 (15), 1456-1468, 2022 | 69 | 2022 |
| Bone health and osteoporosis management of the patient with Duchenne muscular dystrophy LM Ward, S Hadjiyannakis, HJ McMillan, G Noritz, DR Weber Pediatrics 142 (Supplement_2), S34-S42, 2018 | 65 | 2018 |
