Evidence map›Paper›PMID 41461996›Full record

ArticleAdvances in therapy2026

Effectiveness and Safety of Nusinersen Among Adults with 5q-Spinal Muscular Atrophy: A Multicenter Disease Registry in China.

Yi Dai, Xiaoli Yao, Wenhua Zhu, Yuying Zhao, Huifang Shang, Juanjuan Chen, Qiang Meng, Yaling Liu, Wanjin Chen, Liqiang Yu and 6 more

Registry-linked trialAbstract readMulticenter Study
PubMed Publisher
In one paragraph

Article in Advances in therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05618379 (Adult Patients With Spinal Muscular Atrophy in China), which is not on this map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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.

NCT05618379 active not recruitingnot on this map

Adult Patients With Spinal Muscular Atrophy in China: A Nationwide Registry

Typeobservational_patient_registrySponsorBiogenRan2023 to 2028Enrolled200ConditionsMuscular Atrophy, Spinal
3 · Its place in the literature

Who cites it

2 citing papers in PubMed.

  1. Review
  2. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

16 authors.

Yi DaiDepartment of Neurology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Beijing, China.
Xiaoli YaoDepartment of Neurology, The First Affiliated Hospital, Sun Yat-sen University; Guangdong Provincial Key Laboratory of Diagnosis and Treatment of Major Neurological Diseases; National Key Clinical Department and Key Discipline of Neurology, Guangzhou, China.
Wenhua ZhuDepartment of Neurology, Huashan Hospital, Fudan University, Shanghai, China.
Yuying ZhaoDepartment of Neurology, Qilu Hospital of Shandong University, Jinan, China.
Huifang ShangDepartment of Neurology, West China Hospital, Sichuan University, Chengdu, China.
Juanjuan ChenDepartment of Neurology, Peking University Shenzhen Hospital, Shenzhen, China.
Qiang MengDepartment of Neurology, The First People's Hospital of Yunnan Province, Kunming, China.
Yaling LiuDepartment of Neurology, The Second Hospital of Hebei Medical University, ShijiaZhuang, China.
Wanjin ChenDepartment of Neurology, The First Affiliated Hospital of Fujian Medical University, Fuzhou, China.
Liqiang YuDepartment of Neurology, The First Affiliated Hospital of Soochow University, Suzhou, China.
Lu ShenDepartment of Neurology, Xiangya Hospital of Central South University, Changsha, China.
Daojun HongDepartment of Neurology, The First Affiliated Hospital of Nanchang University, Nanchang, China.
Ken ChenReal World Solutions, IQVIA Solutions Enterprise Management Consulting (Shanghai) Co., Ltd, Shanghai, China.
Li ZhangMedical Department, Biogen Biotechnology (Shanghai) Co., Ltd, Shanghai, China.
Maerhaba MaiMedical Department, Biogen Biotechnology (Shanghai) Co., Ltd, Shanghai, China.
Liying CuiDepartment of Neurology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Beijing, China. Cuiliying2025@163.com.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionSpinal muscular atrophy (SMA) is a central nerve system disease characterized by a broad spectrum of clinical presentations, mainly manifested as muscle atrophy and weakness. The effectiveness and safety of nusinersen in Chinese adults with SMA have not been well reported. This analysis aimed to describe the effectiveness and safety of nusinersen among Chinese adults with 5q-SMA.

methodsThis analysis leveraged a longitudinal, multicenter disease registry including both prospective and retrospective data from adults with 5q-SMA in China. Nusinersen's effectiveness was assessed by the changes in the Hammersmith Functional Motor Scale Expanded (HFMSE), Revised Upper Limb Module (RULM), and 6-Minute Walk Test (6-MWT) from baseline and at 6, 10, and 14 months among participants receiving nusinersen as their first disease-modifying therapy (DMT). Nusinersen's safety was evaluated among prospective participants who used nusinersen as their first DMT and initiated nusinersen on or after the enrollment date.

resultsA total of 171 and 19 participants were included in the effectiveness and safety analysis, respectively. For HFMSE, the mean [standard deviation (SD)] change from baseline at 14 months after treatment initiation was 3.4 (5.34) among 17 sitters and 3.4 (4.03) among 25 walkers. For RULM, the mean (SD) change from baseline at 14 months after treatment initiation was 2.6 (3.07) among 11 non-sitters, 1.6 (4.06) among 20 sitters, and 1.3 (1.84) among 13 walkers. For 6-MWT, the mean (SD) change from baseline at 14 months after treatment initiation was 15.0 (35.80) m among 13 walkers. No death was reported in the registry. Three participants reported five mild adverse events, none of which were considered related to nusinersen by the investigators.

conclusionDespite the limited sample size, the analysis appears to show the benefit of nusinersen among Chinese adults of a wide range of ages and disease severity with 5q-SMA. The registry was registered on Clinicaltrials.gov (NCT05618379).

Indexed as

Muscular Atrophy, SpinalOligonucleotidesSpinal Muscular Atrophies of ChildhoodAdultChinaFemaleHumansLongitudinal StudiesMaleMiddle AgedProspective StudiesRegistriesRetrospective StudiesTreatment OutcomeYoung AdultnusinersenOligonucleotidesAdultNusinersenRegistrySpinal muscular atrophy

Identifiers

PMID41461996

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Registered trials

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.