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NEUROMUSCULAR DISEASE

Spinal muscular atrophy — challenges in the therapeutic era

Nature Reviews Neurology volume 16, pages 655–656 (2020)Cite this article

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With the advent of disease-modifying therapies for spinal muscular atrophy, prenatal and extra-neural alterations associated with the condition have garnered increasing attention as potential determinants of the therapeutic window and efficacy of novel drugs. Two recent studies highlight the impact of spinal muscular atrophy on prenatal bone and organ development.

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Fig. 1: Timeline of SMA type 1 pathology and therapeutic options.

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Authors and Affiliations

  1. Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), University of Milan, Milan, Italy

    Irene Faravelli & Stefania Corti

  2. Foundation IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Neurology Unit, Milan, Italy

    Stefania Corti

Authors
  1. Irene Faravelli
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  2. Stefania Corti
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Correspondence to Stefania Corti.

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Competing interests

S.C. has participated in Scientific Advisory Board meetings of AveXis and Scholar Rock and is the Principal Investigator on a no-profit grant from Roche to the University of Milan. I.F declares no competing interests.

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Faravelli, I., Corti, S. Spinal muscular atrophy — challenges in the therapeutic era. Nat Rev Neurol 16, 655–656 (2020). https://doi.org/10.1038/s41582-020-00411-6

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