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Pediatric Spinal Muscular Atrophy (SMA) China Registry
Sponsor: Biogen
Summary
The primary objective of the study is to describe the natural history and utilization of disease modifying therapy (DMT) treatments among pediatric Chinese participants with spinal muscular atrophy linked to chromosome 5q (5q-SMA). The study will examine SMA natural history and DMT outcomes in a real-world setting both prospectively and retrospectively.
Official title: Pediatric Patients With Spinal Muscular Atrophy in China: A Nationwide Registry
Key Details
Gender
All
Age Range
Any - 18 Years
Study Type
OBSERVATIONAL
Enrollment
600
Start Date
2021-11-18
Completion Date
2027-09-02
Last Updated
2025-09-03
Healthy Volunteers
No
Conditions
Locations (25)
Anhui Children's Hospital
Hefei, Anhui, China
Beijing Children's Hospital, Capital Medical University
Beijing, Beijing Municipality, China
Children's Hospital of Chongqing Medical University
Chongqing, Chongqing Municipality, China
Fujian Medical University Union Hospital
Fuzhou, Fujian, China
Xiamen Maternal and Child Health Hospital
Xiamen, Fujian, China
The First Affiliated Hospital, Sun Yat-sen University
Guangzhou, Guangdong, China
Shenzhen Children's Hospital
Shenzhen, Guangdong, China
Maternity and Child Health Care of Guangxi Zhuang Autonomous Region
Nanning, Guangxi Zhuang, China
The Second Hospital of Hebei Medical University
Shijiazhuang, Hebei, China
The Third Affiliated Hospital of Zhengzhou University
Zhengzhou, Henan, China
Hunan Children's Hospital
Changsha, Hu'nan, China
Xiangya Hospital, Central South University
Changsha, Hu'nan, China
Wuhan Children's Hospital, Tongji Medical College Huazhong University of Science & Technology
Wuhan, Hubei, China
The Affiliated Hospital of Inner Mongolia Medical University
Hohhot, Inner Mongolia, China
Xuzhou Central Hospital
Xuzhou, Jiangsu, China
Jiangxi Provincial Children's Hospital
Nanchang, Jiangxi, China
The First Hospital of Jilin University
Changchun, Jilin, China
Children's Hospital Affiliated to Shandong University
Jinan, Jinan Shandong, China
Dalian Women and Children's Medical Group
Dalian, Liaoning, China
Shengjing Hospital of China Medical University
Shenyang, Liaoning, China
People's Hospital of Ningxia Hui Autonomous Region
Yinchuan, Ningxia Hui, China
Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, China
The Second Affiliated Hospital of Xi'An Jiaotong University
Xi’an, Shanxi, China
West China Second University Hospital, Sichuan University
Chengdu, Sichuan, China
The Children's Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, China