Ethical challenges confronted when providing nusinersen treatment for spinal muscular atrophy

Alyssa M. Burgart*, David Magnus, Holly K. Tabor, Erin Daksha Talati Paquette, Joel Frader, Jaqueline J. Glover, Brian M. Jackson, Charlotte H. Harrison, David K. Urion, Robert J. Graham, John F. Brandsema, Chris Feudtner

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

52 Scopus citations


The US Food and Drug Administration’s December 2016 approval of nusinersen for the treatment of patients with all subtypes of spinal muscular atrophy ushered in a new era for patients with spinal muscular atrophy, their families, and all those involved in their care. The extreme cost of the medication and the complicated logistical requirements for administering nusinersen via lumbar puncture have created practical challenges that raise important ethical considerations. We discuss 6 challenges faced at the institutional level in the United States: cost, limited evidence, informed consent, treatment allocation, fair distribution of responsibilities, and transparency with stakeholders. These challenges must be understood to ensure that patients with spinal muscular atrophy benefit from treatment, are protected from harm, and are treated fairly.

Original languageEnglish (US)
Pages (from-to)188-192
Number of pages5
JournalJAMA Pediatrics
Issue number2
StatePublished - Feb 1 2018

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health


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