Evidence map›Paper›PMID 40758201›Full record

ArticleWorld journal of pediatrics : WJP2025

Single-dose GC101 gene therapy for spinal muscular atrophy types II and III: an open-label single-arm study.

Xiu-Wei Ma, Xin-Yang Jiang, Zhong-Qiu Li, Xiao-Yan Dong, Wen-Hao Ma, Yong-Xia Wang, Shuang-Qing Yu, Rui-Jie Gu, Xiao-Dong Wang, Bai-Mao Zhong and 16 more

Abstract read
In one paragraph

Article in World journal of pediatrics : WJP, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the 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.

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

2 citing papers in PubMed.

  1. Article
  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

26 authors.

Xiu-Wei MaDepartment of Pediatrics, Faculty of Pediatrics, The Seventh Medical Center of the Chinese PLA General Hospital, Beijing 100700, China.
Xin-Yang JiangDepartment of Pediatrics, Faculty of Pediatrics, The Seventh Medical Center of the Chinese PLA General Hospital, Beijing 100700, China.
Zhong-Qiu LiDepartment of Pediatrics, Faculty of Pediatrics, The Seventh Medical Center of the Chinese PLA General Hospital, Beijing 100700, China.
Xiao-Yan DongGenecradle Therapeutics Inc., Beijing 100176, China.
Wen-Hao MaGenecradle Therapeutics Inc., Beijing 100176, China.
Yong-Xia WangDepartment of Pediatrics, Faculty of Pediatrics, The Seventh Medical Center of the Chinese PLA General Hospital, Beijing 100700, China.
Shuang-Qing YuGenecradle Therapeutics Inc., Beijing 100176, China.
Rui-Jie GuDepartment of Pediatrics, Faculty of Pediatrics, The Seventh Medical Center of the Chinese PLA General Hospital, Beijing 100700, China.
Xiao-Dong WangGenecradle Therapeutics Inc., Beijing 100176, China.
Bai-Mao ZhongDepartment of Pediatrics, Dongguan Children's Hospital, Guangdong 523325, China.
Fang HeDepartment of Pediatrics, Faculty of Pediatrics, The Seventh Medical Center of the Chinese PLA General Hospital, Beijing 100700, China.
Juan XuDepartment of Pharmacy, Medical Supplies Center of the Chinese PLA General Hospital, Beijing 100700, China.
Ying-Ying MaoGenecradle Therapeutics Inc., Beijing 100176, China.
Yan-Ping ZhangDepartment of Pediatrics, Faculty of Pediatrics, The Seventh Medical Center of the Chinese PLA General Hospital, Beijing 100700, China.
Shan ZhangDepartment of Pediatrics, Faculty of Pediatrics, The Seventh Medical Center of the Chinese PLA General Hospital, Beijing 100700, China.
Ting LiDepartment of Pediatrics, Faculty of Pediatrics, The Seventh Medical Center of the Chinese PLA General Hospital, Beijing 100700, China.
Chong-Yang LiDepartment of Pediatrics, Faculty of Pediatrics, The Seventh Medical Center of the Chinese PLA General Hospital, Beijing 100700, China.
Sheng ZhangDepartment of Pediatrics, Faculty of Pediatrics, The Seventh Medical Center of the Chinese PLA General Hospital, Beijing 100700, China.
Xiao YangDepartment of Pediatrics, Faculty of Pediatrics, The Seventh Medical Center of the Chinese PLA General Hospital, Beijing 100700, China.
Li-Na ZhuDepartment of Pediatrics, Faculty of Pediatrics, The Seventh Medical Center of the Chinese PLA General Hospital, Beijing 100700, China.
Zhi-Jie WuGenecradle Therapeutics Inc., Beijing 100176, China.
Bing ZhouAdvanced Innovation Center for Big Data-Based Precision Medicine, Beihang University, Beijing 100191, China.
Lu ZhuangDepartment of Pediatrics, Faculty of Pediatrics, The Seventh Medical Center of the Chinese PLA General Hospital, Beijing 100700, China.
Qiu-Ping LiDepartment of Pediatrics, Faculty of Pediatrics, The Seventh Medical Center of the Chinese PLA General Hospital, Beijing 100700, China.
Xiao-Bing WuGenecradle Therapeutics Inc., Beijing 100176, China. wuxiaobing@bj-genecradle.com.
Zhi-Chun FengDepartment of Pediatrics, Faculty of Pediatrics, The Seventh Medical Center of the Chinese PLA General Hospital, Beijing 100700, China. zhichunfeng81@163.com.ORCID 0000-0002-9622-4062

Funding

E-Town Cooperation & Development Foundation YCXJ-JZ-2022-007
6 · The paper itself

Abstract

QUICK TAKE.

Indexed as

Genetic TherapySpinal Muscular Atrophies of ChildhoodChildChild, PreschoolFemaleHumansInfantMaleTreatment Outcome

Identifiers

PMID40758201
PMCPMC12433340

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.