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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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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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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DOI: https://doi.org/10.1038/s41582-020-00410-7
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