Spinal Muscular Atrophy
Conditions
Interventions
Sponsors
Eligibility
Inclusion criteria
Inclusion criteria: 1. Diagnosis by a neurologist through a genetic laboratory test that verifies a homozygous deletion of exon 7 (with or without exon 8) and 3 SMN2 copy sites in patients with type III SMA; 2. Applicants should be between the ages of 3 and 17 years old, and the cognitive ability level of the study participants should be considered, and whether they could understand and cooperate with the experiment; 3. Patients with SMAIII type can walk independently or walk more than 10 meters with an orthosis/walker; 4. Study participants have been routinely treated with Nusinersen to ensure consistency of assessments; 5. Outpatient physiotherapy at least 5 times a week; 6. All study participants or legal guardians understand the study and sign the informed consent form.
Exclusion criteria
Exclusion criteria: 1. Patients who have previously undergone spinal surgery and require prolonged bedside care; 2. Study participants participate in other rehabilitation treatment programs related to SMA in hospitals, clinics or rehabilitation centers; 3. There are other conditions that are not suitable for rehabilitation exercises; 4. Study participant or legal guardian unable to sign informed consent; 5. Study participants can only participate in less than 5 treatments per week for various reasons; 6. Study participants are unable to complete the study due to fatigue or muscle weakness.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Hammersmith functional motor scale Expanded,HFMSE; | — |
Secondary
| Measure | Time frame |
|---|---|
| Manual Muscle Testing, MMT;6-Minute Walk Test;10-Minute Walk Test/Shuttle Run Test;Pediatric Quality of Life Inventory Multi Dimensional Fatigue Score,PedsQLMFS;Adverse events; | — |
Countries
China
Contacts
West China Hospital, Sichuan University