Anna Mayhew

Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

Craig M. McDonald, Craig Campbell, Ricardo Erazo Torricelli et al. · The Lancet · 2017 · 446 citations · Full text

Rare Diseases, Urology, Clinical Trials +5

Revised upper limb module for spinal muscular atrophy: Development of a new module

Elena Mazzone, Anna Mayhew, Jacqueline Montes et al. · Muscle & Nerve · 2016 · 342 citations

Long-term benefits and adverse effects of intermittent versus daily glucocorticoids in boys with Duchenne muscular dystrophy

Valeria Ricotti, Deborah Ridout, E. Scott et al. · Journal of Neurology Neurosurgery & Psychiatry · 2012 · 255 citations

Patterns of disease progression in type 2 and 3 SMA: Implications for clinical trials

Eugenio Mercuri, Richard S. Finkel, Jacqueline Montes et al. · Neuromuscular Disorders · 2015 · 212 citations · Full text

Disease Progression, Clinical Epidemiology, Chronic Disease +9

Content validity and clinical meaningfulness of the HFMSE in spinal muscular atrophy

Maria Carmela Pera, Giorgia Coratti, Nicola Forcina et al. · BMC Neurology · 2017 · 209 citations · Full text