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Treating adults with spinal muscular atrophy with nusinersen
  1. Michelle A Farrar1,2,
  2. Matthew C Kiernan3
  1. 1Neurology, Sydney Children's Hospital, Randwick, New South Wales, Australia
  2. 2School of Women's and Children's Health, UNSW Medicine, UNSW Sydney, Sydney, New South Wales, Australia
  3. 3Brain 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

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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.

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