Stopped: Due to the lack of application materials, the normal research can not be carried out for the time being.
The primary objectives of this study are to obtain clinically meaningful data on the survival, outcomes, prognosis and treatment effect of all the patients with spinal muscular atrophy (SMA) 5q types 1 to 3 (according to international classification), being followed in the Children's Hospital, Zhejiang University School of Medicine since October 2019. The registry will collect retrospectively and prospectively the longitudinal data of the long-term follow-up for children patients, under real life conditions of current medical practice, in order to document the clinical evolution of patients (survival, motor, respiratory, orthopedic and nutritional and so on), the conditions of use of the treatments, the mortality rates of treated and untreated patients, the tolerance of the treatments, adverse events.
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
World Health Organization (WHO) motor milestone change
Timeframe: baseline, 0.5 year, 1 year, 1.5 year, 2 year, 2.5 year, 3 year
Hammersmith Infant Neurological Examination-2 (HINE-2) scale score change
Timeframe: baseline, 0.5 year, 1 year, 1.5 year, 2 year, 2.5 year, 3 year
Self-reporting/Caregivers' reporting collection
Timeframe: baseline, 0.5 year, 1 year, 1.5 year, 2 year, 2.5 year, 3 year
Motor function evalution-Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders(CHOP INTEND) scale
Timeframe: treated/untreated: baseline, 0.5 year, 1 year, 1.5 year, 2 year, 2.5 year, 3 year
Motor function evalution-Hammersmith Functional Motor Scale-Expanded(HFMSE) scale
Timeframe: treated/untreated: baseline, 0.5 year, 1 year, 1.5 year, 2 year, 2.5 year, 3 year
Motor function evalution-Revised Upper Limb Module(RULM) scale
Timeframe: treated/untreated: baseline, 0.5 year, 1 year, 1.5 year, 2 year, 2.5 year, 3 year
Motor function evalution change-6 minutes walking test(6MWT)
Timeframe: treated/untreated: baseline, 0.5 year, 1 year, 1.5 year, 2 year, 2.5 year, 3 year
Yearly changes of incidence and morbi-mortality-vital events
Timeframe: every year for 3 years
Change from baseline respiratory function
Timeframe: baseline(before treatment), 1 year, 2 year, 3 year
Change from baseline digestive-nutritional function
Timeframe: baseline(before treatment), 0.5 year, 1 year, 1.5 year, 2 year, 2.5 year, 3 year
Change from baseline spinal scoliosis Cobb angle
Timeframe: baseline(before treatment), 1 year, 2 year, 3 year