{
  "abstract": "Spinal muscular atrophy (SMA) is characterized by motor neuron degeneration due to deficiency in the survival of motor neuron (SMN) protein. Disease-modifying therapies have transformed outcomes in SMA, while also revealing an increasingly complex spectrum of residual disease biology. In this issue of the JCI, Tapken, Rahmel-Stein, and colleagues demonstrated cell-specific SMN expression and persistent alterations in peripheral immune cell populations in nusinersen- or risdiplam-treated adults with SMA. Their findings extend the question of selective vulnerability beyond motor neurons and raise an increasingly important therapeutic question: What constitutes adequate SMN restoration? This Commentary considers how cell type, developmental timing, SMN requirement, and downstream response may shape treatment-modified SMA, with implications for systemic and CNS-directed therapies.",
  "abstract_shown": true,
  "affiliations": [
    "National Neuroscience Institute, Singapore, Singapore.",
    "Institute of Molecular and Cell Biology (IMCB), Agency for Science, Technology and Research (A*STAR), Singapore, Singapore.",
    "Duke-National University of Singapore Medical School, Singapore, Singapore.",
    "Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA."
  ],
  "article_type": "article commentary",
  "arxiv": null,
  "author_total": 1,
  "authors": [
    {
      "affs": [
        0,
        1,
        2,
        3
      ],
      "name": "Crystal Jing Jing Yeo"
    }
  ],
  "bibtex": "https://research.draconitic.com/paper/62fws687ajvje.bib",
  "cited_by": 0,
  "doi": "10.1172/jci211991",
  "fulltext_url": "https://europepmc.org/articles/PMC13722389",
  "id": "62fws687ajvje",
  "issns": [
    "0021-9738",
    "1558-8238"
  ],
  "landing_url": "https://europepmc.org/article/MED/42969230",
  "language": "eng",
  "licence": "CC-BY",
  "licence_label": "CC BY",
  "licence_source": "article",
  "licence_url": "https://creativecommons.org/licenses/by/4.0/",
  "metadata_licence": null,
  "native_subjects": [
    [
      "heading",
      "Commentary"
    ],
    [
      "openalex",
      "2716|Neurogenetic and Muscular Disorders Research"
    ]
  ],
  "openalex": "W7225062358",
  "pdf_url": "https://europepmc.org/articles/PMC13722389?pdf=render",
  "pmcid": "PMC13722389",
  "pmid": "42969230",
  "published": "2026-11-02",
  "publisher": "American Society for Clinical Investigation",
  "ris": "https://research.draconitic.com/paper/62fws687ajvje.ris",
  "sources": [
    {
      "first_seen": "2026-11-03T15:20:53Z",
      "native_id": "MED:42969230",
      "source": "europepmc",
      "url": "https://europepmc.org/article/MED/42969230"
    },
    {
      "first_seen": "2026-11-04T12:22:00Z",
      "native_id": "PMC13722389",
      "source": "pmc",
      "url": "https://doi.org/10.1172/jci211991"
    }
  ],
  "subjects": [
    {
      "field": "medicine",
      "origin": "openalex:2716|Neurogenetic and Muscular Disorders Research",
      "subfield": "human-genetics"
    }
  ],
  "title": "Beyond motor rescue: defining adequate SMN restoration in spinal muscular atrophy.",
  "title_text": "Beyond motor rescue: defining adequate SMN restoration in spinal muscular atrophy.",
  "type": "peer-reviewed",
  "updated_at": "2026-11-05T12:20:12Z",
  "url": "https://research.draconitic.com/paper/62fws687ajvje",
  "venue": "The Journal of clinical investigation",
  "versions": []
}