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Editorial commentary
Treating adults with spinal muscular atrophy with nusinersen
  1. Michelle A Farrar1,2,
  2. Matthew C Kiernan3
  1. 1 Neurology, Sydney Children's Hospital, Randwick, New South Wales, Australia
  2. 2 School of Women's and Children's Health, UNSW Medicine, UNSW Sydney, Sydney, New South Wales, Australia
  3. 3 Brain and Mind Centre, The University of Sydney, Sydney, New South Wales, Australia
  1. Correspondence to Dr Michelle A Farrar, Neurology, Sydney Children's Hospital Randwick, Randwick NSW 2031, Australia; m.farrar{at}unsw.edu.au

https://doi.org/10.1136/jnnp-2020-324036

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    Intrathecal nusinersen to treat adults with spinal muscular atrophy can be administered safely and with clinically meaningful results.

    Nusinersen was the first drug approved to treat people with spinal muscular atrophy (SMA), based on clinical data obtained from the initial trials undertaken in infants and children.1 2 With limited evidence of the safety and efficacy of nusinersen across the spectrum of ages and severities in SMA, adult patients are now confronting uncertain therapeutic expectations, with associated difficulties in realising equitable access to treatment.3 4 In the paper by Maggi and colleagues5 in this issue of the journal, further evidence indicates that treatment with nusinersen can be administered safely to adult patients with SMA, with clinically meaningful results obtained in a ‘real world’ setting.

    This Italian multicentre study included a …

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    Footnotes

    • Twitter @imichellefarrar

    • Contributors MAF and MK planned, drafted and revised the manuscript. All authors read and approved the submitted version.

    • Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.

    • Competing interests None declared.

    • Patient consent for publication Not required.

    • Provenance and peer review Commissioned; internally peer reviewed.

    Linked Articles

    • Neuromuscular
      Nusinersen safety and effects on motor function in adult spinal muscular atrophy type 2 and 3
      Lorenzo Maggi Luca Bello Silvia Bonanno Alessandra Govoni Claudia Caponnetto Luigia Passamano Marina Grandis Francesca Trojsi Federica Cerri Manfredi Ferraro Virginia Bozzoni Luca Caumo Rachele Piras Raffaella Tanel Elena Saccani Megi Meneri Veria Vacchiano Giulia Ricci Gianni Soraru' Eustachio D'Errico Irene Tramacere Sara Bortolani Giovanni Pavesi Riccardo Zanin Mauro Silvestrini Luisa Politano Angelo Schenone Stefano Carlo Previtali Angela Berardinelli Mara Turri Lorenzo Verriello Michela Coccia Renato Mantegazza Rocco Liguori Massimiliano Filosto Gianni Marrosu Gabriele Siciliano Isabella Laura Simone Tiziana Mongini Giacomo Comi Elena Pegoraro
      Journal of Neurology, Neurosurgery & Psychiatry 2020; 91 1166-1174 Published Online First: 11 Sep 2020. doi: 10.1136/jnnp-2020-323822