Duchenne muscular dystrophy
Conditions
Interventions
Sponsors
Eligibility
Inclusion criteria
Inclusion criteria: 1) Presence of antimyotrophic protein gene mutation diagnosed by genetic examination and/or presence of decreased or absent antimyotrophic protein expression confirmed by muscle biopsy; 2) Male children between the ages of 6 and 12 years; 3) Ambulatory; 4) The measured value of MIP was 30%-90% of the predicted value or the measured value of FVC was 30%-90% of the predicted value, where the predicted value of MIP (cmH2O) = 153 + (-1.27 × age) + (0.131 × body weight), and the predicted value of FVC (L) = 0.93 × 1.1 × ((0.0576 × height) - (0.0269 × age) - 4.34); 5) Can cooperate to complete active rehabilitation training; 6) Written informed consent signed by the child or the child's family.
Exclusion criteria
Exclusion criteria: 1) Lung infection and lung injury within the last 3 months; 2) Combination of other neurodevelopmental disorders (e.g. autism spectrum disorders, language disorders, etc.); 3) Comorbidity with other neuromuscular disorders (e.g., dermatomyositis, myasthenia gravis, peripheral neuropathy, etc.); 4) Those with a combination of severe mood disorders, aggressive behavior, and oppositional defiance; 5) Those who withdraw from or fail to complete the treatment in the middle of the program.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Spirometry; | — |
Secondary
| Measure | Time frame |
|---|---|
| Six-minute walking test (6MWT);End-tidal carbon dioxide pressure (EtCO2);North Star Ambulatory Assessment (NSAA);respiratory muscle strength;Functional timing test (10 m run/walk test);Anthropometric indicators; | — |
Countries
China
Contacts
West China Second University Hospital