Hugh J. McMillan

Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy

Deepa H. Chand, F. Mohr, Hugh J. McMillan et al. · Journal of Hepatology · 2020 · 297 citations · Full text

Medicine, Pharmacology, Toxicology +17

Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial

Kevin A. Strauss, Michelle A. Farrar, Francesco Muntoni et al. · Nature Medicine · 2022 · 295 citations · Full text

Medicine, Genetics, Pediatrics +13

Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial

Kevin A. Strauss, Michelle A. Farrar, Francesco Muntoni et al. · Nature Medicine · 2022 · 276 citations · Full text

Medicine, Genetics, Pediatrics +16

Myostatin inhibitor ACE‐031 treatment of ambulatory boys with Duchenne muscular dystrophy: Results of a randomized, placebo‐controlled clinical trial

Craig Campbell, Hugh J. McMillan, Jean K. Mah et al. · Muscle & Nerve · 2016 · 234 citations

Pharmacy, Pharmacokinetics, Pharmacology +14

Newborn screening programs for spinal muscular atrophy worldwide: Where we stand and where to go

Tamara Dangouloff, Eva Vrščaj, Laurent Servais et al. · Neuromuscular Disorders · 2021 · 172 citations

Medicine, Pediatrics, Preventive Medicine +12