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April 15, 2026JAMA Pediatrics0 citations

Out-of-Pocket Cost of Spinal Muscular Atrophy Gene Therapies Among Commercially Insured Patients

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MSMaria SckaffIOIlina C. OdouardJBJeromie Ballreich

Key Points

  • To assess the insurance coverage and out-of-pocket costs associated with spinal muscular atrophy gene therapies.
  • Cross-sectional study design
  • Evaluation of commercial insurance data
  • Analysis of patient spending from 2021-2022
  • Overall out-of-pocket costs for patients using SMA therapies were substantial
  • Variations in costs were observed across different therapies
  • Insurance coverage significantly influenced patient expenditure

Abstract

This cross-sectional study evaluates insurance and patient spending for the spinal muscular atrophy medications nusinersen, onasemnogene abeparvovec-xioi, and risdiplam from 2021 through 2022.

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Cite This Study

Sckaff et al. (2026) studied this question.

synapsesocial.com/papers/69df2cb9e4eeef8a2a6b1ffchttps://doi.org/10.1001/jamapediatrics.2026.0641
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Drug treatment for spinal muscular atrophy types II and III2012 · 100 citations
  2. 2Evaluation of muscle strength and motor abilities in children with type II and III spinal muscle atrophy treated with valproic acid2011 · 44 citations
  3. 3Triptolide increases transcript and protein levels of survival motor neurons in human SMA fibroblasts and improves survival in SMA‐like mice2012 · 24 citations
  4. 4Coverage of genetic therapies for spinal muscular atrophy across fee-for-service Medicaid programs2021 · 8 citations
  5. 5Drug treatment for spinal muscular atrophy type I2019 · 27 citations