The primary objective of the study is to describe the natural history and utilization of disease modifying therapy (DMT) among adult Chinese participants with SMA linked to chromosome 5q (5q-SMA).
Study Type
OBSERVATIONAL
Enrollment
200
Peking Union Medical College Hospital
Beijing, Beijing Municipality, China
The First Affiliated Hospital of Fujian Medical University
Fuzhou, Fujian, China
The First Affiliated Hospital, Sun Yat-sen University
Guangzhou, Guangdong, China
Number of Participants Characterized by Natural History of 5q-SMA Observed Among Adult Chinese Participants
The natural history characteristics will include the categories of the evolution of the patient's condition over the time of data collection from the registry, to describe the natural history of the disease in a real-life situation.
Time frame: Up to 60 Months
Number of Participants Characterized by Utilization of Disease Modifying Therapies (DMTs) of 5q-SMA Observed Among Adult Chinese Participants
The DMT utilization characteristics will include the categories of the evolution of the DMT treated patient's condition over the time of data collection from the registry, to describe the utilization of DMTs of adult SMA patients in a real-life situation.
Time frame: Up to 60 Months
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Peking University Shenzhen Hospital
Shenzhen, Guangdong, China
The Second Hospital of Hebei Medical University
Shijiazhuang, Hebei, China
Xiangya Hospital of Central South University
Changsha, Hunan, China
The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, China
The First Affiliated Hospital of Nanchang University
Nanchang, Jiangxi, China
Qilu Hospital of Shandong University
Jinan, Shandong, China
Huashan Hospital, Fudan University
Shanghai, Shanghai Municipality, China
...and 2 more locations