Evidence map›Paper›PMID 39960588›Full record

SynthesisAdvances in therapy2025

Motor Function and Safety of Nusinersen and Risdiplam in Asian Patients with Types 2-4 Spinal Muscular Atrophy (SMA): A Systematic Review and Meta-Analysis.

Xinran Zhao, Yihan Liao, Jingyu Zhao, Lin Zhu, Jun Liu, Min Zhang, Wei Li

Abstract readMeta-AnalysisSystematic Review
PubMed Publisher
In one paragraph

Synthesis in Advances in therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
7citing papers in PubMed, 1 pooled it
–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

7 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Review
  3. Article
  4. Risdiplam therapy in adult patients with Spinal muscular Atrophy (SMA). A 24-month-real-world experience at a single muscular centre.Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2026
    Article
  5. Article
  6. Review
  7. Article
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

7 authors.

Xinran Zhao *IQVIA China Real-World Solutions, Shanghai, 200124, China.
Yihan Liao *IQVIA China Real-World Solutions, Shanghai, 200124, China.
Jingyu ZhaoIQVIA China Real-World Solutions, Shanghai, 200124, China.
Lin ZhuBiogen China Value and Access, Shanghai, 200031, China.
Jun LiuIQVIA China Real-World Solutions, Shanghai, 200124, China.
Min ZhangShanxi Bethune Hospital, Shanxi Academy of Medical Sciences, Third Hospital of Shanxi Medical University, Tongji Shanxi Hospital, Taiyuan, Shanxi, 030032, China. zhang_min_3464@126.com.
Wei LiChina Pharmaceutical University, Nanjing, Jiangsu, 211198, China. cpuliwei@163.com.ORCID 0000-0003-2164-9266

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionThe aim of this study was to systematically appraise and synthesize real-world data of motor function and safety in Asian patients with spinal muscular atrophy (SMA) treated with nusinersen or risdiplam.

methodsThis study systematically searched PubMed, Cochrane, Embase, CNKI, and Wanfang databases for real-world studies (RWS) published from January 2017 to January 2024. Based on the prespecified study selection and eligibility criteria, RWS evaluating motor function and/or safety outcomes in patients with types 2-4 SMA treated with nusinersen or risdiplam were included, while studies without Asian populations were excluded. The Newcastle-Ottawa Scale (NOS) was used to assess the risk of bias, and a meta-analysis was conducted for each motor function endpoint based on the extracted data.

resultsA total of 26 RWS were included in this review, of which 17 reporting main motor function outcomes were included in the meta-analyses. Intervention in all 17 studies was nusinersen; none included risdiplam. Statistically significant improvement was observed in Revised Upper Limb Module (RULM) [Mean difference (MD) = 2.27 (0.84, 3.71)], Hammersmith Functional Motor Scale Expanded (HFMSE) [MD = 2.62 (1.79, 3.45)] and six-minute walk test (6MWT) [MD = 18.29 (9.12, 27.45)] when treated with nusinersen ≤ 6 months and > 6 months (HFMSE [MD = 4.34 (3.54, 5.14)]; 6MWT [MD = 45.59 (12.92, 78.27)]). Clinically meaningful responses of motor milestones were also observed when treating nusinersen over 6 months: 54.4% (0.305, 0.772) for RULM, 53.0% (0.273, 0.779) for HFMSE and 97.1% (0.819, 1.000) for 6MWT. A total of 19 studies addressed safety outcomes. Reported adverse events were consistent with the expected safety profile for nusinersen.

conclusionOur study suggests that nusinersen is associated with a statistically significant and clinically meaningful motor function improvement in Asian patients with types 2-4 SMA in the real-world setting. No unexpected safety concern was observed for nusinersen. Motor function and safety outcomes after risdiplam could not be evaluated in this patient population under real-world settings due to limited studies.

Indexed as

Muscular Atrophy, SpinalOligonucleotidesPyrimidinesSpinal Muscular Atrophies of ChildhoodAsian PeopleAzo CompoundsDrug CombinationsHumansAzo CompoundsDrug CombinationsnusinersenOligonucleotidesPyrimidinesRisdiplamAsiaMeta-analysisMotor functionNusinersenRisdiplamSafetySpinal muscular atrophySystematic review

Identifiers

PMID39960588

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

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.