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Avindra Nath MD
Avindra Nath MD Associate Editor
Argye Hillis MD, MA
Argye Elizabeth Hillis MD, MA Associate Editor
Mark Hallett MD
Mark Hallett MD Editor-in-Chief
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Published in Neurology

Expert Opinion / Commentary · November 27, 2017

2017 Top Stories in Neurology: New Therapeutics in Spinal Muscular Atrophy

Marinos Dalakas MD, FAAN

 

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Additional Info

  1. Finkel RS, Mercuri E, Darras BT, et al. Nusinersen versus sham control in infantile-onset spinal muscular atrophy. N Engl J Med. 2017;377(18):1723-1732.
  2. van der Ploeg AT. The dilemma of two innovative therapies for spinal muscular atrophy. N Engl J Med. 2017;377(18):1786-1787.
  3. Mendell JR, Al-Zaidy S, Shell R, et al. Single-dose gene-replacement therapy for spinal muscular atrophy. N Engl J Med. 2017;377(18):1713-1722.

Disclosure statements are available on the authors' profiles:

Marinos Dalakas MD, FAAN

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