Evidence map›Paper›PMID 41706311›Full record

ArticleActa neurologica Belgica2026

Clinical outcomes and efficacy of nusinersen in Korean adult patients with 5q spinal muscular atrophy: a nationwide multicenter retrospective study.

Jong-Mok Lee, Hyung Jun Park, Hyunjin Kim, Ha Young Shin, Young-Eun Park, Minsung Kang, Yeon Hak Chung, Ju-Hong Min, Jinhong Shin

Erratum issuedAbstract readMulticenter Study
PubMed Publisher
In one paragraph

Article in Acta neurologica Belgica, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

9 authors.

Jong-Mok LeeDepartment of Neurology, School of Medicine, Kyungpook National University, Kyungpook National University Hospital, Daegu, South Korea.ORCID http://orcid.org/0000-0002-2918-6166
Hyung Jun ParkDepartment of Neurology, Gangnam Severance Hospital, Yonsei University College of Medicine, Seoul, Republic of Korea.ORCID http://orcid.org/0000-0003-4165-8901
Hyunjin KimDepartment of Neurology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea.ORCID http://orcid.org/0000-0003-0264-4531
Ha Young ShinDepartment of Neurology, Yonsei University College of Medicine, Seoul, Republic of Korea.ORCID http://orcid.org/0000-0002-4408-8265
Young-Eun ParkDepartment of Neurology, Pusan National University School of Medicine, Busan, Korea.ORCID http://orcid.org/0000-0003-0887-1604
Minsung KangDepartment of Neurology, Kyungpook National University Chilgok Hospital, Daegu, South Korea.ORCID http://orcid.org/0000-0001-6206-0891
Yeon Hak ChungDepartment of Neurology, Korea University Guro Hospital, Korea University College of Medicine, Seoul, Korea.ORCID http://orcid.org/0000-0002-7052-2432
Ju-Hong MinDepartment of Neurology, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, South Korea.ORCID http://orcid.org/0000-0002-7338-9067
Jinhong ShinDepartment of Neurology, Pusan National University Yangsan Hospital, Pusan National University School of Medicine, Yangsan, Republic of Korea. shinzh@gmail.com.ORCID http://orcid.org/0000-0002-5174-286X

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionSpinal muscular atrophy (SMA) is a genetic motor neuron disorder, marked by progressive skeletal and respiratory muscle weakness. The recent approval of nusinersen has transformed the therapeutic landscape for SMA patients, particularly children. However, as randomized controlled studies and long-term real-world evidence in adults remain limited, this study focuses on the efficacy of nusinersen in adults with SMA in South Korea.

methodClinical data were retrospectively collected from adult patients who began nusinersen in tertiary neurology centers across the country, with 19 patients eventually being enrolled.

resultsThe results showed that Hammersmith Functional Motor Scale-Expanded scores improved significantly by 2.28 (p = 0.0002) in all patients after 12 months. Following the dropout of 4 patients who failed to meet insurance reimburse criteria, improvements of 4.55 (p = 0.0010) and 6.57 (p = 0.0156) points were noted in the remaining 15 patients after 24 and 36 months, respectively. At these time points, 40%, 82%, and 86% of patients, respectively, accomplished clinically meaningful improvements. Type 2 SMA patients exhibited a faster drug response compared to type 3a patients. Out of 176 injections, seven adverse events were reported, including post-puncture headache and injection site pain.

conclusionsOur multicenter real-world study demonstrates the sustained efficacy and manageable safety profile of nusinersen in adult SMA patients, supporting the long-term use in this population.

Indexed as

Muscular Atrophy, SpinalOligonucleotidesAdolescentAdultFemaleHumansMaleMiddle AgedRepublic of KoreaRetrospective StudiesTreatment OutcomeYoung AdultnusinersenOligonucleotidesAxonsMotor neuron diseasesOligonucleotide antisense

Identifiers

PMID41706311

What OpenQuestion holds

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.