Spinal Muscular Atrophy
Conditions
Interventions
Trial Group:WeChat App-based remote rehabilitation program (including breathing exercises, trunk control training, balance training, and mobility training), performed at home for no less than 5 times
Control Group:Standard home-based rehabilitation (home exercise program based on Chinese expert consensus and international guidelines for multidisciplinary management of spinal muscular atrophy, incl
Sponsors
West China Hospital of Sichuan University
Eligibility
Sex/Gender
All
Age
3 Years to 17 Years
Inclusion criteria
Inclusion criteria: 1. Inclusion Criteria for SMA Type III Participants: All of the following conditions must be met: (1) Genetically confirmed diagnosis of spinal muscular atrophy type III, with homozygous deletion of exon 7 (with or without deletion of exon 8) and three copies of the SMN2 gene; (2) Age between 3 and 17 years (this range is set based on disease subtype characteristics and cognitive capacity considerations); (3) Ability to walk at least 10 meters independently or with orthoses/walking aids; (4) Initiation of nusinersen treatment at the study hospital to ensure standardized follow-up assessments by the same physical therapy team; (5) Thorough understanding of the trial objectives, procedures, etc., and signed informed consent obtained from the patient or their legal guardian. 2. Inclusion Criteria for SMA Type II Participants: All of the following conditions must be met: (1) Diagnosis of SMA type II confirmed by a neurologist through genetic laboratory testing; (2) Age between 3 and 17 years, primarily considering the cognitive ability of participants to understand and cooperate with the trial, with Mini-Mental State Examination (MMSE) score = 27; (3) Ability to maintain sitting balance with assistance or independently (meeting Bobath sitting balance scale level I or below level I); (4) Participants are already receiving routine nusinersen treatment to ensure consistency in assessments; (5) Thorough understanding of the trial objectives, procedures, etc., and signed informed consent obtained from the patient or their legal guardian.
Exclusion criteria
Exclusion criteria: 1. Exclusion Criteria for SMA Type III Participants: Patients will be excluded if they meet any of the following criteria: (1) History of spinal surgery requiring prolonged bed rest; (2) Current participation in other specialized rehabilitation programs targeting spinal muscular atrophy; (3) Inability to receive the intervention of either the experimental or control group due to other medical reasons; (4) Inability of the participant or their parent/guardian to provide signed informed consent; (5) Inability to complete the study due to fatigue or muscle weakness. 2. Exclusion Criteria for SMA Type II Participants: Patients will be excluded if they meet any of the following criteria: (1) History of spinal surgery requiring prolonged bedside care; (2) Current participation in other SMA-related rehabilitation treatment programs at a hospital, clinic, or rehabilitation center; (3) Presence of other conditions unsuitable for rehabilitation exercise, such as pain during movement, post-exercise injury, or muscle strain; (4) Inability of the participant or their legal guardian to provide signed informed consent; (5) Inability to complete the study due to fatigue or muscle weakness.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Hammersmith functional motor scale Expanded; | — |
Secondary
| Measure | Time frame |
|---|---|
| Pediatric Quality of Life Inventory-Chinese Version;Functional Independence Measure for Children; | — |
Countries
China
Contacts
Public ContactZhang Jing
West China Hospital of Sichuan University
Outcome results
None listed