P62 The identification of a viable outcome measure in the collagen VI myopathies promotes progress toward clinical trials

Abstract

1FC2.1 Exon skipping and dystrophin restoration in Duchenne Muscular Dystrophy patients after systemic phosphorodiamidate morpholino oligomer treatment S. Cirak1 *, V. Arechavala-Gomeza1, M. Guglieri2, L. Feng1, S. Torelli1, K. Anthony1, M.E. Garralda3, D.J. Wells4, G. Dickson5, M.J.A. Wood6, S. Wilton7, V. Straub2, S.B. Shrewsbury8, C. Sewry9, J.E. Morgan1, K. Bushby2, F. Muntoni1. 1UCL Institute of Child Health The Dubowitz Neuromuscular Centre, London, United Kingdom, 2Institute of Human Genetics, Newcastle University, Newcastle, United Kingdom, 3Academic Unit of Child and Adolescent Psychiatry, Imperial College St Mary’s Campus, London, United Kingdom, 4Royal Veterinary College, London, United Kingdom, 5Royal Holloway University of London, United Kingdom, 6Department of Physiology, Anatomy and Genetics, University of Oxford, United Kingdom, 7Centre for Neuromuscular and Neurological Disorders, University of Western Australia, Perth, Australia, 8AVI BioPharma, Bothell, WA, United States, 9Centre for Inherited Neuromuscular Disorders, Robert Jones and Agnes Hunt NHS Trust, Oswestry, United Kingdom