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Spinal muscular atrophy — the dawning of a new era

Nature Reviews Neurology volume 16, pages 593–594 (2020)Cite this article

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The introduction of therapies for spinal muscular atrophy (SMA) has rapidly changed the clinical landscape, transforming SMA from a lethal to a treatable disease. This transformation has driven further advances, from population screening imperatives to novel treatment delivery approaches, while uncovering health disparities and fuelling debate regarding drug pricing.

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Fig. 1: The evolving landscape of spinal muscular atrophy.

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Acknowledgements

M.A.F. was supported by a Motor Neurone Diseases Research Institute of Australia Innovator Grant (1G 1916). M.C.K. was supported by a National Health and Medical Research Council of Australia (NHMRC) Practitioner Fellowship (1156093). Funding from the NHMRC Program Grant (#1132524), Dementia Research Team Grant (#1095127) and Partnership Project (1153439) are gratefully acknowledged.

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Authors and Affiliations

  1. Discipline of Paediatrics, School of Women’s and Children’s Health, UNSW Medicine, UNSW Sydney, Sydney, New South Wales, Australia

    Michelle A. Farrar

  2. Brain & Mind Centre and Sydney Medical School, University of Sydney, Sydney, New South Wales, Australia

    Matthew C. Kiernan

  3. Department of Neurology, Royal Prince Alfred Hospital, Sydney, New South Wales, Australia

    Matthew C. Kiernan

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  1. Michelle A. Farrar
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Correspondence to Michelle A. Farrar.

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Competing interests

M.A.F. has received compensation as a member of the scientific advisory boards for Biogen, Roche and AveXis. These bodies had no role in the design or preparation of the article or decision to publish. M.C.K. is Editor-in-Chief of the Journal of Neurology, Neurosurgery & Psychiatry (BMJ Publishers, UK).

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Farrar, M.A., Kiernan, M.C. Spinal muscular atrophy — the dawning of a new era. Nat Rev Neurol 16, 593–594 (2020). https://doi.org/10.1038/s41582-020-00410-7

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