Emerging Therapies and Challenges in Spinal Muscular Atrophy

Michelle A Farrar; Susanna B Park; Steve Vucic; Kate A Carey; Bradley J Turner; Thomas H Gillingwater; Kathryn J Swoboda; Matthew C Kiernan

Journal article

Emerging Therapies and Challenges in Spinal Muscular Atrophy

Michelle A Farrar, Susanna B Park, Steve Vucic, Kate A Carey, Bradley J Turner, Thomas H Gillingwater, Kathryn J Swoboda, Matthew C Kiernan

Annals of Neurology | WILEY | Published : 2017

DOI: 10.1002/ana.24864

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University of Melbourne Researchers

Bradley Turner Author Florey Department of Neuroscience and Mental Health

Related Projects (1)

SMN IMMUNOGENE THERAPY FOR MOTOR NEURON DISORDERS

We have designed novel gene therapy agents to treat MND. This therapeutic approach uniquely combines gene therapy agents with antibodies whi..

Grants

Awarded by National Health and Medical Research Council

Awarded by National Institutes of Child Health and Development from the National Institutes of Health

Awarded by EUNICE KENNEDY SHRIVER NATIONAL INSTITUTE OF CHILD HEALTH & HUMAN DEVELOPMENT

Funding Acknowledgements

M.F. received support from the Motor Neurone Diseases Research Institute of Australia Beryl Bayley MND Postdoctoral Fellowship. T.G. received support from the UK SMA Research Consortium (SMA Trust) and Muscular Dystrophy UK. B.T. received support from the National Health and Medical Research Council project grant (#1104299), Stafford Fox Medical Research Foundation, and Cure for MND Foundation. K.J.S. received support from the National Institutes of Child Health and Development grant R01-HD69045 from the National Institutes of Health.