Matthew Harmelink

Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial

Craig M. McDonald, Eduardo Marbán, Suzanne Hendrix et al. · The Lancet · 2022 · 78 citations · Full text

Siblings With Mutations in TRAPPC11 Presenting With Limb-Girdle Muscular Dystrophy 2S

Dominic B. Fee, Matthew Harmelink, Priya Monrad et al. · Journal of Clinical Neuromuscular Disease · 2017 · 40 citations

Rare Diseases, Developmental Biology, Mendelian Disorder +14

Assessment of cerebral spinal fluid biomarkers and microRNA-mediated disease mechanisms in spinal muscular atrophy patient samples

Emily Welby, Rebecca Rehborg, Matthew Harmelink et al. · Human Molecular Genetics · 2021 · 22 citations

Pediatric Opsoclonus-Myoclonus-Ataxia Syndrome Associated With Anti-N-methyl-D-aspartate Receptor Encephalitis

Brittany Player, Matthew Harmelink, Brett J. Bordini et al. · Pediatric Neurology · 2015 · 15 citations

Neurological Disorder, Medicine, Neurology +4

Outcomes of early‐treated infants with spinal muscular atrophy: A multicenter, retrospective cohort study

Natalie L. Goedeker, Amanda Rogers, Mark Fisher et al. · Muscle & Nerve · 2024 · 10 citations · Full text